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Alabama Chapter
President: Rhyann Granger
P.O. Box 71918  Tuscaloosa,  AL  35407
P: (205) 979-6493  E: alabama@fsma.org

Visit our online SMA Community by clicking here. First, login or create an account. Then, click the orange "Groups" tab at the top of the page.  Scroll through until you find "Alabama Chapter" and click "Join Group" to start connecting with other families and friends in your area.

Chapter Events
August 23, 2014   7th Annual Macy's Shop for a Cause
June 12, 2014   The 2014 Annual SMA Conference
November 2, 2013   11th Annual Alabama Chapter Walk-n-Roll to Cure SMA
Local Chapter News
July 29, 2014   Stop SMA Charity Auction
Our friends at Stop SMA are doing an online auction where you can bid on valuable items and know that the proceeds help stop SMA! If you have any questions or would like to donate an item, contact Jeff Horton by sending an email to president@stopsma.org. Click the link for more information!
August 3, 2013   Laugh Away SMA
Join Stop SMA on August 3rd, 2013 at the Muse Center at the Hinds Rankin campus in Raymond, MS to Laugh Away SMA! Featured comedians include Henry Cho John Heffron, and Jake Gulledge. Click the link above for information about how to purchase tickets. Visit www.charityauctionstoday.com/store/stopsma to see the online auction!
December 26, 2012   Logan Patrick Passes Away
Logan Patrick passed away on Saturday, December 22nd, 2012. Please click the link above for more information and an article about Logan's fight with SMA.
December 1, 2012   Country Christmas with Skylar Laine
Join Stop SMA on December 1, 2012 for Country Christmas with Skylar Laine at the Jackson Academy Performing Arts Center in Jackson, MS! Tickets are available at www.STOPSMA.org. Click the link above for more details.
August 4, 2012   Laugh Away SMA
Join Stop SMA for the Laugh Away SMA comedy show featuring Henry Cho and John Reep on Saturday, August 4 at 7 p.m. at the Muse Center on Hinds Community College's Rankin Campus. Visit www.laughawaysma.org for more details!
National News
August 20, 2014   Families of Spinal Muscular Atrophy Awards Second Year of Research Funding to Dr. Lyndsay Murray for a Total of $80,000.
FSMA will be committing $1.8 million to new research funding over the coming year.
 
August 18, 2014   Thank You to the FSMA Medical Advisory Council and Scientific Advisory Board for a Great Spinal Muscular Atrophy Conference
At our Annual SMA Conference in June, we asked families to write short notes of thanks to our Medical Advisory Council and Scientific Advisory Board for all the hard work they’ve put into planning the conference.
 
August 15, 2014   NeuroNEXT Recruitment Update: 25 infants with SMA Have Enrolled in the Study with 2 More Infants Needed.
 
August 15, 2014   Call Congress and Urge Passage of The ABLE Act
 
August 14, 2014   Families of SMA provides critical services to families recently diagnosed with Spinal Muscular Atrophy
 
August 12, 2014   SMA Researchers Talk About the Importance of the Spinal Muscular Atrophy Research Group Meeting
 
August 11, 2014   Photos Posted for Annual SMA Candle Lighting for Spinal Muscular Atrophy Awareness Month
 
August 8, 2014   Join Families of SMA at the Ballpark to Raise Awareness for Spinal Muscular Atrophy
 
August 7, 2014   Spinal Muscular Atrophy Mouse Model Results Published in the Journal Science
 
August 6, 2014   Update from Dr. Lorson at University of Missouri on Spinal Muscular Atrophy Program being Funded by Families of SMA
 
August 5, 2014   Using Social Media to Reach Out During Spinal Muscular Atrophy Awareness Month
 
August 1, 2014   Isis Initiates Phase 3 Clinical Trial in Infants with Spinal Muscular Atrophy.
 
July 31, 2014   Help Fund a Cure for Spinal Muscular Atrophy – Over 65 Fall Fundraisers to Choose From
 
July 30, 2014   2014 Spinal Muscular Atrophy Awareness Month: What You Can Do to Help Develop a Cure for SMA
 
July 25, 2014   Collaborative Study to Understand Patient Perspectives on Meaningful Clinical Trial Benefit to Continue in Cities Across The United States.
Families of SMA, the SMA Foundation, and several industry partners are co-funding this research project to better understand which clinical trial outcomes are particularly meaningful to SMA patients.
 
July 25, 2014   U.S. Senate Committee Encourages NIH to Continue SMA-Related Research Initiatives
 
July 22, 2014   Families of Spinal Muscular Atrophy Releases a New Care Series Booklet for Families about The Musculoskeletal System
 
July 16, 2014   Families of SMA Releases a New Request for Proposals for Clinical Care Research Projects in Spinal Muscular Atrophy
 
July 11, 2014   National Institutes of Health Releases Three Grant Opportunities Related to Newborn Screening for Disease like Spinal Muscular Atrophy.
These grant opportunities respond to years of report language endorsed by Families of SMA in the annual congressional appropriations bills that funds the NIH.
 
July 10, 2014   Families of Spinal Muscular Atrophy Continuing Medical Education Conference Presentations Now Available
 
July 9, 2014   Families of SMA Releases a Request for Proposals for New Funding of Research Projects for Spinal Muscular Atrophy.
Families of SMA is pleased to announce that we are accepting grant applications for funding of research projects, under a competitive review by our Scientific Advisory Board. Up to $700,000 will be committed in this round of funding.
 
July 7, 2014   Attendee Video from The 2014 Annual Spinal Muscular Atrophy Conference Online.
 
June 27, 2014   Families of Spinal Muscular Atrophy Announces Two Winners of The 2014 Annual SMA Conference iPad Survey Giveaway.
 
June 27, 2014   Families of Spinal Muscular Atrophy Adaptive Arts Project for The 2014 Annual SMA Conference a Huge Success!
The Jacob Isaac Rappoport Foundation awarded Families of SMA with funding for an Adaptive Arts Project for the Annual SMA Conference which was a huge hit with all the children who attended!
 
June 27, 2014   Families of SMA Releases Keynote Speeches and Drug Companies Summaries from The 2014 Annual Spinal Muscular Atrophy Conference
 
June 26, 2014   Location Announced for The 2015 Annual SMA Conference.
 
June 26, 2014   AveXis Announces Dosing of First Patient for Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1.
Click here for more information and for the latest update presented by AveXis at the Annual SMA Conference.
 
June 26, 2014   Family Friendly Research Poster Session at The 2014 Annual Spinal Muscular Atrophy Conference.
 
June 26, 2014   Tirasemtiv Demonstrates Improvements in Muscle Force, Grip Strength and Resistance to Fatigue in Preclinical Mouse Models of Spinal Muscular Atrophy
 
June 25, 2014   U.S. House of Representatives Approves Newborn Screening Legislation: Includes Provisions Advocated by Families of Spinal Muscular Atrophy.
 
June 23, 2014   Families of Spinal Muscular Atrophy Thanks our Exhibitors for Attending The 2014 Annual SMA Conference in National Harbor, MD.
 
June 17, 2014   Motor Function Testing (MFT) in the NeuroNEXT Infant SMA Biomarker Study
The study investigators are recruiting infants who do not have SMA less than 1 year of age from the Chicagoland area to be videotaped at a session at Ann & Robert H. Lurie Children’s Hospital of Chicago.
 
June 9, 2014   The 2014 Annual Spinal Muscular Atrophy Conference Special Events and Exciting Facts.
 
June 5, 2014   Families of Spinal Muscular Atrophy Continuing Medical Education Conference on Friday, June 13th
 
June 5, 2014   Families of Spinal Muscular Atrophy New England Chapter Cure SMA Walk-n-Roll Reaches $1,000,000 Milestone.
 
June 5, 2014   Presenters Announced for The Family and Researcher Poster Session at The Annual Spinal Muscular Atrophy Conference.
Over thirty posters will be presented during the session.
 
June 4, 2014   Advocacy Activities at The 2014 Annual Spinal Muscular Atrophy Conference in Washington, D.C.
 
June 3, 2014   Collaborative Study to Understand Patient Perspectives on Meaningful Clinical Trial Benefit to be Initiated at The Annual SMA Conference.
Families of SMA, the SMA Foundation, and several industry partners are co-funding this research project to better understand which clinical trial outcomes are particularly meaningful to SMA patients.
 
May 29, 2014   Families of Spinal Muscular Atrophy Thanks the Sponsors for The 2014 Annual SMA Conference.
Families of SMA is pleased to announce that we have 25 Sponsors registered for the upcoming 2014 Annual SMA Conference.
 
May 28, 2014   Families of SMA Awarded Grant from the National Institute of Health for the 2014 Spinal Muscular Atrophy Research Group Meeting.
 
May 28, 2014   Families of Spinal Muscular Atrophy Michigan Chapter Run, Walk-n-Roll Reaches Milestone 10th Annual Year
 
May 27, 2014   Families of Spinal Muscular Atrophy Thanks our Registered Exhibitors for The 2014 Annual SMA Conference in National Harbor, MD.
Families of SMA is excited to announce that we have 25 registered exhibitors for The 2014 Annual SMA Conference!
 
May 23, 2014   Conference Booklet and Detailed Agenda for The 2014 Annual Spinal Muscular Atrophy Conference Now Available Online.
This 88 page booklet includes the detailed agenda of the 42 conference workshops, with information on each speaker, as well as all events and conference details.
 
May 23, 2014   Genzyme Publishes Paper on Spinal Muscular Atrophy Gene Therapy Program with Intrathecal Delivery.
Genzyme scientists will be presenting data this June at the Annual SMA Conference in the Research Q&A Session and the Family Friendly Research Poster Session.
 
May 15, 2014   Registration for the 2014 SMA Research Group Meeting is Open until May 28.
 
May 13, 2014   Families of Spinal Muscular Atrophy Receives 200 Bath Kneeler Pads for the Newly Diagnosed SMA Care Package Program in Memory of Joseph Lillo.
 
May 12, 2014   House Energy & Commerce Committee Initiates 21st Century Cures with 1st Roundtable
 
May 8, 2014   Annual Spinal Muscular Atrophy Conference Registration Deadline is Only One Day Away!
 
May 1, 2014   Registration for The 2014 Annual Spinal Muscular Atrophy Conference Exceeds 1,200.
 
April 29, 2014   Isis Presents Update on Spinal Muscular Atrophy Drug Trials at the American Academy of Neurology Meeting.
This includes results from two open-label Phase 2 clinical studies of ISIS-SMNRx in infants and children with SMA. They are on track to start Phase III trials later in 2014.
 
April 29, 2014   Trophos Presents Pivotal Phase II/III Study Results on Olesoxime in Spinal Muscular Atrophy at The American Academy of Neurology Meeting.
 
April 25, 2014   Phase I Clinical Trial of Systemic SMN Gene Therapy For Spinal Muscular Atrophy is Open at Nationwide Children’s Hospital.
 
April 24, 2014   Families of Spinal Muscular Atrophy is Currently Funding 24 Research Projects with Multiple Rounds of New Funding to be Announced in 2014.
Later in 2014 Requests for Proposals (RFPs) with new funding announcements will be made for over $1.5 Million.
 
April 23, 2014   New Edition of Compass Newsletter on Spinal Muscular Atrophy Clinical Care Research Grants.
 
April 15, 2014   Update on Families of Spinal Muscular Atrophy Funding to The California Institute of Biomedical Research
Developing new therapies for SMA is the driving force behind Families of SMA funding strategy. To achieve this goal, Families of SMA funds successful and innovative scientists like Dr. Peter Schultz.
 
April 10, 2014   Families of Spinal Muscular Atrophy Thanks our Sponsors for The 2014 Annual SMA Conference.
Families of SMA is pleased to announce that we currently have 18 sponsors registered for the upcoming 2014 Annual SMA Conference.
 
April 9, 2014   Families of Spinal Muscular Atrophy Awards New Research Funding of $50,000 to Dr. Lyndsay Murray.
Lyndsay Murray of The University of Edinburgh will explore the reasons causing motor neuron cell death in SMA.
 
April 8, 2014   Families of Spinal Muscular Atrophy Announces Panelists for The Research Question and Answer Session at The 2014 Annual SMA Conference.
Leading experts will address new advances, strategies, and challenges in SMA drug development.
 
April 7, 2014   Families of SMA Spring Fundraising Event Season is Finally Here to Help Fund a Cure for Spinal Muscular Atrophy.
Thanks to so many amazing Chapter leaders and SMA Families, we are aiming to raise over $2.1 Million to fund advancements in SMA research and critical family support and patient care programs.
 
April 2, 2014   Families of Spinal Muscular Atrophy is Thrilled to Announce that Registration for The 2014 Annual SMA Conference Has Now Exceeded 1,000
With just 5 weeks remaining until registration closes on May 8th, the 2014 Annual SMA Conference, held on June 12th – June 15th, will be a conference not to miss!
 
April 1, 2014   Regenx Biosciences and Avexis Enter into Agreement for The Development of Treatments for Spinal Muscular Atrophy Using NavRaav9 Vectors
The license is important for the successful development and commercialization of AAV9 based gene therapy for SMA.
 
April 1, 2014   Families of Spinal Muscular Atrophy Awards New Research Funding of $75,000 to Dr. Chien-Ping Ko.
Dr. Chien-Ping Ko at The University of Southern California will explore the role of glial cells in SMA.
 
March 27, 2014   NIH Holds SO-SMART Workshop: Significant Outcomes-Spinal Muscular Atrophy (SMA) Readies for Trials.
On June 11, the day prior to the Annual SMA Conference in Washington DC, the NIH is hosting a workshop on clinical trial design in SMA.
 
March 25, 2014   Families of Spinal Muscular Atrophy Awards New Research Funding of $100,000 to Dr. Rashmi Kothary.
Dr. Rashmi Kothary at The Ottawa Hospital Research Institute will receive $100,000 to explore non-SMN mediated mechanisms to treat SMA.
 
March 19, 2014   Families of Spinal Muscular Atrophy Awards New Research Funding of $95,000 to Dr. Kejun Han.
Dr. Ke-Jun Han at the University of Colorado will explore the transport of SMN protein into motor axons.
 
March 13, 2014   Workshops Announced for The 2014 Annual SMA Conference.
 
March 12, 2014   The Pennington Foundation Provides Opportunities for Medical Professionals to Attend the FSMA 2014 Continuing Medical Education Conference.
 
March 12, 2014   Do you have a child with Spinal Muscular Atrophy? Has your child participated in a clinical trial within the past 10 years?
We invite you to take part in a survey to understand how the neuromuscular community thinks and feels about potential therapeutics and clinical trials.
 
March 11, 2014   Families of Spinal Muscular Atrophy Awards New Research Funding of $95,000 to Dr. Allison Ebert.
Dr. Ebert at the Medical College of Wisconsin will explore the role of neuronal support cells called astrocytes in SMA.
 
March 10, 2014   Trophos Announces Top-line Results of Pivotal Trial of Olesoxime in Spinal Muscular Atrophy Showing Promise for Maintaining Motor Function in SMA.
 
March 6, 2014   100 Research Presentations at The 2014 Spinal Muscular Atrophy Research Group Meeting Hosted and Supported by Families of SMA.
The meeting is the largest SMA research conference in the world. Multiple events are planned for researchers and families to interact.
 
March 4, 2014   Families of Spinal Muscular Atrophy Awards New Research Funding of $95,000 to Dr. Barrington Burnett.
Dr. Burnett of the Uniformed Services University of the Health Sciences will explore the role of SMN in muscle development.
 
February 26, 2014   Families of SMA Equipment Pool Grows after Donation from Jadon’s Hope Foundation.
Jadon’s Hope Foundation made a purchase of much needed equipment in November of 2013.
 
February 24, 2014   Families of Spinal Muscular Atrophy Awards New Research Funding of $90,000 to Drs. Bogdanik and Lutz at The Jackson Laboratory.
They will work in collaboration with Dr. W. David Arnold at The Ohio State University and Dr. Seward Rutkove at Harvard Medical School to explore electrophysiological endpoints in drug testing in mice.
 
February 24, 2014   Families of Spinal Muscular Atrophy Plans Family and Researcher Events at The 2014 SMA Conference.
 
February 21, 2014   ISIS Pharmaceuticals Reports Interim Results on Current Trials in Spinal Muscular Atrophy
 
February 21, 2014   The Miller McNeil Woodruff Foundation Partners with Families of SMA to Provide Scholarships for The 2014 Continuing Medical Education Conference.
 
February 20, 2014   Families of Spinal Muscular Atrophy Awards $600,000 for Basic Research to Identify New Approaches for SMA Therapies.
In the new issue of the newsletter “Compass”, Families of SMA announces our seven basic research grant awards.
 
February 18, 2014   Sweet Baby Zane Donates 30 Medical Provider Scholarships for The Families of Spinal Muscular Atrophy 2014 Continuing Medical Education Conference.
 
February 13, 2014   Update as Isis Pharmaceuticals Advances ISIS-SMN Rx in Infants and Children with Spinal Muscular Atrophy
 
February 12, 2014   Families of Spinal Muscular Atrophy Receives 200 Touch and Feel Baby Books for the Newly Diagnosed SMA Care Package Program in Memory of Logan Ruth.
 
February 10, 2014   Lukie’s Fall Festival Reaches Milestone Year Raising Funds for Families of SMA
 
January 31, 2014   Start a Fundraiser With These Resources and Ideas From Families of SMA!
The Families of SMA spring event season is already making huge progress toward our goal of raising over $1.6 million to fund exciting new Spinal Muscular Atrophy research and critical family support programs.
 
January 30, 2014   Senate Approves Newborn Screening Legislation. Next Step is House Action to Establish Foundation for Adding SMA to Federal Newborn Screening Panel.
 
January 22, 2014   PTC Therapeutics and Roche Announce Spinal Muscular Atrophy Drug Enters First Stage of Clinical Development.
This brings the total number of SMA programs in clinical development to 5.
 
January 20, 2014   Families of Spinal Muscular Atrophy Offers Valuable Scholarship Opportunity for the Continuing Medical Education Conference on SMA.
 
January 17, 2014   Thank You for Giving Back Over $550,000 for Spinal Muscular Atrophy Research.
Thank you for sharing your strength. Thank you for all you gave back.
 
January 15, 2014   Families of SMA Announces 2014 Legislative Agenda and Exciting Advocacy Opportunities. The Families of SMA Day on Capitol Hill.
Our legislative advocacy efforts will culminate with “The Families of SMA Day on Capitol Hill” on June 11, 2014, the day before the official start of The Annual SMA Conference.
 
January 10, 2014   Registration for The 2014 Continuing Medical Education Conference on SMA Held on June 13th Now Available.
 
January 8, 2014   Spinal Muscular Atrophy Study Opportunity From The Children’s Hospital of Philadelphia.
 
December 31, 2013   Families of Spinal Muscular Atrophy Annual Campaign Reaches $500,000 for SMA Research.
 
December 30, 2013   Families of Spinal Muscular Atrophy Contributes Research Funding to Sixteen SMA Journal Articles in 2013.
FSMA has invested over $55 Million in research to find a treatment and cure for SMA, including 25 research projects in 2013 alone.
 
December 28, 2013   Families of Spinal Muscular Atrophy Annual Campaign Over Halfway to Goal.
 
December 27, 2013   SMA Takes Away Mobility. You Give Back Action.
 
December 19, 2013   Families of Spinal Muscular Atrophy Provides Record Number of Resources and Support to SMA Families Worldwide.
Along with funding SMA research, Families of SMA provides thousands of families with vital family support and resources that help families navigate life with SMA.
 
December 18, 2013   Double Your Support For Newly Diagnosed SMA Families
 
December 18, 2013   Families of Spinal Muscular Atrophy Scientific Advisory Board Meets To Evaluate Basic SMA Research Projects for New Funding.
This week our SAB met in Chicago to select the best projects for funding. Total funding of up to $700,000 will be committed to research projects, which will be announced in January 2014.
 
December 17, 2013   SMA Community Mourns the Passing of David Cunniff.
 
December 16, 2013   Families of SMA Releases Version of Family Guide to Spinal Muscular Atrophy Research Translated in Spanish.
Families of SMA has just released the Spanish version of this booklet, which covers the basics of Spinal Muscular Atrophy research for SMA families.
 
December 13, 2013   Opportunities for Involvement in Clinical Studies for SMA Patients and Families.
 
December 12, 2013   The Ins and Outs of Antisense Drugs for Spinal Muscular Atrophy.
This newsletter was produced in a collaboration between Families of SMA, Biogen Idec, and Isis Pharmaceuticals.
 
December 3, 2013   Families of Spinal Muscular Atrophy 2014 Annual Campaign. Help us make sure no promising SMA research goes unfunded.
 
December 2, 2013   Families of Spinal Muscular Atrophy Continuing Medical Education Conference at The 2014 Annual SMA Conference in Washington, DC.
 
November 26, 2013   SMA Takes Away. You Give Back Strength.
 
November 25, 2013   New Edition of the Families of Spinal Muscular Atrophy Directions Newsletter.
The Fall 2013 Directions Newsletter is now available.
 
November 22, 2013   Isis Pharmaceuticals to Investigate a Higher Dose of ISIS-SMNRx in Children with Spinal Muscular Atrophy
 
November 19, 2013   2nd Annual “Hope on the Hill” Congressional Dinner to Raise Funds and Awareness for Spinal Muscular Atrophy.
 
November 15, 2013   Families of Spinal Muscular Atrophy Announces the Schedule for The 2014 Annual SMA Conference.
The schedule is announced for the 2014 Annual SMA Conference held June 12th – June 15th, 2014 in Washington, DC.
 
November 13, 2013   Spinal Muscular Atrophy Researchers Participate at The 2013 Society of Neuroscience Meeting.
The Society of Neuroscience (SfN) Meeting is the premiere neurobiology meeting with 30,000 attending. Multiple presentations on SMA will be given by FSMA-funded researchers, as well as other prominent scientists.
 
November 4, 2013   Families of Spinal Muscular Atrophy Receives Basic Research Grant Applications for New Funding.
The best projects will be selected for funding by our Scientific Advisory Board in Chicago this December.
 
October 30, 2013   Survey Published of 24 SMA National Registries, Mapping the Differences in Care for 5,000 Spinal Muscular Atrophy Patients.
Researchers analyzed the TREAT-NMD national patient SMA registries in order to provide a snapshot of data by SMA subtype and compared the results.
 
October 25, 2013   Scholarships Are Now Available for The 2014 Annual SMA Conference.
 
October 24, 2013   BAYADA Pediatrics Renews Sponsorship of the Families of SMA Walk-n-Roll Program.
 
October 24, 2013   Families of Spinal Muscular Atrophy Funds 25 Research Projects in 2013.
FSMA invests in 4 research areas: Basic Research, Drug Discovery, Clinical Research, and Care Research.
 
October 21, 2013   The NeuroNEXT Infant Biomarker Study for Spinal Muscular Atrophy Reaches Recruitment Milestone.
The spots for the infants without SMA have been fulfilled by 27 “Super Families.” The study has also enrolled 12 infants with SMA. They are still looking for 15 more infants with SMA to enroll.
 
October 17, 2013   The Research Institute at Nationwide Children’s Hospital Receives Fast Track Status for Spinal Muscular Atrophy Treatment.
This is third SMA program to receive Fast Track designation from the FDA! In addition, AveXis licenses patent portfolio from Nationwide Children's.
 
October 10, 2013   Isis Provides FSMA an Update on Clinical Development Plans for Spinal Muscular Atrophy
 
October 9, 2013   Journal of Child Neurology Publishes on Nutritional Practices at a Glance: Spinal Muscular Atrophy Type I Nutrition Survey Findings
 
October 8, 2013   Congratulations to Susi Vander Wyk, President of Families of SMA Canada, Recently Announced as Walmart Canada’s 2013 Mom of the Year.
 
October 1, 2013   Meet Newly Funded Families of SMA Researcher Dr. Timothy Lotze.
Dr. Timothy Lotze was awarded funding by Families of SMA for one of four new Clinical Care focused projects. The project is being conducted at Texas Children’s Hospital at the Baylor College of Medicine.
 
September 25, 2013   Meet Newly Funded Families of SMA Researcher Dr. Matthew Halanski
Dr. Halanski was awarded funding by Families of SMA for one of four new clinical care focused projects.
 
September 20, 2013   FDA Approval to Nationwide Children’s Hospital for Phase I Clinical Trial of Systemic AAV9-Delivered SMN Gene For Spinal Muscular Atrophy
The clinical trial is expected to begin in early 2014 and will be limited to Type I SMA patients, ages birth to 9 months.
 
September 19, 2013   Isis Reports Follow-Up Data From Phase 1 Study In Children With Spinal Muscular Atrophy.
 
September 17, 2013   Meet Newly Funded Families of Spinal Muscular Atrophy Researcher: Dr. Kathryn Swoboda.
Dr. Swoboda was awarded funding by Families of SMA for one of four new Clinical Care focused projects.
 
September 10, 2013   Meet Newly Funded Families of SMA Researchers: Oscar Mayer, MD PhD and Vanessa Battista, MS.
They were awarded funding by Families of SMA for one of four new clinical care focused projects.
 
September 5, 2013   SMA Families Increase Awareness of Spinal Muscular Atrophy Throughout the Month of August
 
September 4, 2013   Pfizer RG3039 Program for Spinal Muscular Atrophy Reaches Development Milestone
Reaching the first milestone was triggered by completion of specific program activities and coincides with the successful completion of all transition obligations to Pfizer from Repligen.
 
September 3, 2013   Help Fund a Cure for Spinal Muscular Atrophy – Over 50 Fall Fundraisers to Choose From
 
August 30, 2013   Registration for The 2014 Annual SMA Conference at The Gaylord National is Now Available.
 
August 29, 2013   Families of SMA Releases a Request for Proposals for New Funding of Research Projects for Spinal Muscular Atrophy.
Families of SMA is pleased to announce that we are accepting grant applications for new funding of research projects, under a competitive review by our Scientific Advisory Board (SAB).
 
August 28, 2013   Families of Spinal Muscular Atrophy Scientific Advisory Board Highlights Findings from The 2013 SMA Research Group Meeting.
 
August 22, 2013   Meet Newly Funded Families of SMA Researcher: Dr. Yimin Hua.
Dr. Hua is a Research Investigator at Cold Spring Harbor Laboratory in the laboratory of Dr. Adrian Krainer. He is a long-term collaborator of Isis Pharmaceuticals.
 
August 21, 2013   New Edition of the Compass Newsletter on The 2013 Spinal Muscular Atrophy Research Conference
In this edition of Compass hear from leading SMA scientists about how the meeting fosters mentorship, collaboration, and integration into the SMA research community.
 
August 19, 2013   Record Breaking Fundraising Events Year in Support of Families of Spinal Muscular Atrophy.
 
August 15, 2013   Meet Newly Funded Families of SMA Researcher: Dr. Adrian Krainer
Dr. Krainer is a Professor at Cold Spring Harbor Laboratory, a member of the FSMA Scientific Advisory Board, and a long-term collaborator with Isis Pharmaceuticals.
 
August 13, 2013   New Edition of Compass Newsletter Released on the Spinal Muscular Atrophy Drug Pipeline
This newsletter provides an update on the 2013 SMA Drug Pipeline and describes new FSMA funding to Dr. Rubin at Harvard University and to Cytokinetics.
 
August 12, 2013   Photos Posted of Annual SMA Candle Lighting for Spinal Muscular Atrophy Awareness Month.
Many photos taken of children and families during the Annual SMA Candle Lighting are now posted on the Families of SMA Facebook Page.
 
August 9, 2013   PTC Therapeutics Announces Selection of Development Candidate for Spinal Muscular Atrophy
 
August 8, 2013   Families of SMA Awards $150,000 to Drs. Hua and Krainer at CSHL to Investigate Antisense Oligonucleotides for Spinal Muscular Atrophy
The fourth new drug project funded by Families of SMA in 2013.
 
August 7, 2013   Join Families of SMA at the Ballparks to Raise Awareness for Spinal Muscular Atrophy
Several Major League Baseball teams are teaming up with Families of SMA to help raise awareness and funds for finding a treatment and a cure for Spinal Muscular Atrophy.
 
August 7, 2013   Project to Optimize Testing Measures for Spinal Muscular Atrophy Clinical Trials.
 
August 6, 2013   Families of Spinal Muscular Atrophy Supports Newly Introduced Bipartisan Newborn Screening Bill in the Sentate
U.S. Senators Kay Hagan (D-NC) and Orrin Hatch (R-UT) introduced the bipartisan Newborn Screening Saves Lives Reauthorization Act.
 
August 1, 2013   Families of SMA’s Second Continuing Medical Education Conference for Medical Professionals
 
July 31, 2013   Presentations from The 2013 Annual Spinal Muscular Atrophy Conference Workshops Now Available Online.
Presentations from some of the 40 workshops offered during the conference covering the latest information on critical topics in SMA Care are now available on the FSMA website.
 
July 30, 2013   Photos from The 2013 Annual Spinal Muscular Atrophy Conference Are Now Available.
 
July 29, 2013   Videos of Research Question and Answer Session at The 2013 SMA Conference Released by Families of Spinal Muscular Atrophy
Leading experts on SMA drug development answer questions about their drug programs.
 
July 26, 2013   Families of Spinal Muscular Atrophy Thanks our Exhibitors for Attending The 2013 Annual SMA Conference in Anaheim, CA.
Families of SMA was thrilled to have 30 Exhibitors attend the 2013 Annual SMA Conference.
 
July 24, 2013   10th Annual Pennsylvania Chapter Cure SMA Walk-n-Roll Reaches Milestone Year
 
July 22, 2013   Families of Spinal Muscular Atrophy Defending the Orphan Drug Tax Credit
 
July 22, 2013   Posters Available from the 1st Family Friendly SMA Research Poster Session at The Annual Spinal Muscular Atrophy Conference
Twenty-three research groups, including those representing ten SMA drug programs, presented their scientific findings.
 
July 19, 2013   2013 SMA Awareness Month: What You Can Do with Support From Families of Spinal Muscular Atrophy
August is SMA Awareness month and families and friends around the country are joining together to help increase awareness of this devastating disease.
 
July 18, 2013   Recruitment Update on the NeuroNEXT Trial on Spinal Muscular Atrophy Biomarkers in the Immediate Postnatal Period of Development Study
Trial PI, Dr. Stephen Kolb of Ohio State University, presented information about the study at the 2013 SMA Conference.
 
July 17, 2013   Highlights from The 2013 Annual SMA Conference Meet and Greet.
 
July 11, 2013   Families of Spinal Muscular Atrophy Provides Summaries of Drug Programs Represented at The 2013 SMA Conference.
 
July 10, 2013   Families of Spinal Muscular Atrophy Releases Keynote Speeches from the Research Question and Answer Session at The 2013 Annual SMA Conference.
The 2013 SMA Conference brought together 1,300 families and researchers from around the world. At the conclusion of the conference families gathered for a special opportunity to hear about the latest SMA research.
 
June 26, 2013   SMA Families and Chapters Make The Annual Spinal Muscular Atrophy Conference Possible
 
June 26, 2013   Attendee Video from The 2013 Annual Spinal Muscular Atrophy Conference Online.
 
June 25, 2013   Families and Researchers Interact at The 2013 Annual Spinal Muscular Atrophy Conference.
225 researchers from 15 different countries, 70 distinct organizations, and 17 biotech and pharmaceutical companies attended the 2013 SMA meeting.
 
June 25, 2013   Amy Marquez Scholarship Announced at The 2013 Annual Spinal Muscular Atrophy Conference.
 
June 24, 2013   New Funding of SMA Patient Care Research Announced at The Annual Spinal Muscular Atrophy Conference.
 
June 21, 2013   Families of Spinal Muscular Atrophy Adaptive Arts Project for The 2013 Annual SMA Conference a Huge Success!
 
June 21, 2013   Families of Spinal Muscular Atrophy Awards Two Winners for The 2013 Annual SMA Conference iPad Survey Giveaway
 
June 20, 2013   Location Announced for The 2014 Annual SMA Conference
 
June 19, 2013   The Jacob Isaac Rappoport Foundation Marks Incredible Milestone at The 2013 Annual SMA Conference.
In an amazing demonstration of their generosity and dedication the family presented an incredible donation check for $100,000!
 
June 7, 2013   Families of Spinal Muscular Atrophy Announces Expected Attendance for The 2013 Annual SMA Conference Over 1,300 with Participants from 23 Countries.
The 2013 Annual SMA Conference on June 13th to June 16th, 2013 at The Disneyland Hotel in Anaheim, CA has closed registration with over 1,300 attendees from all over the world.
 
June 7, 2013   Families of Spinal Muscular Atrophy is excited to Launch the 2013 Annual SMA Conference App for your Mobile Device.
The 2013 Annual SMA Conference App is your personal touch point for essential event information, such as the conference agenda, workshop descriptions, speaker biographies, exhibitor lists, a map of the venue and much more.
 
June 4, 2013   Two Studies Testing the Drug RG3039 in Multiple Mouse Models of Spinal Muscular Atrophy Published in Human Molecular Genetics.
FSMA invested $13 million for the preclinical development of RG3039. Repligen licensed RG3039 from FSMA in 2009. In January 2013, Pfizer licensed the program, marking a significant advance for the SMA community by securing the commitment of one of the largest pharmaceutical companies to SMA. It is currently being tested in Phase 1B human clinical trials.
 
May 31, 2013   Families of SMA Awarded Grant from the National Institute of Health for the 2013 Spinal Muscular Atrophy Research Group Meeting.
Families of SMA has received a grant from the National Institute of Neurological Disorders and Disease (NINDS) at the National Institutes for Health (NIH). We thank them for their support that helps bring together the SMA research community along with our families.
 
May 31, 2013   Families of Spinal Muscular Atrophy Thanks our Amazing Sponsors for the upcoming 2013 Annual SMA Conference.
Families of SMA is pleased to announce that we currently have 19 Sponsors supporting the upcoming 2013 Annual SMA Conference.
 
May 23, 2013   Conference Booklet and Detailed Agenda for the 2013 Annual Spinal Muscular Atrophy Conference Now Available Online.
This 80 page booklet includes the detailed agenda of the 44 conference workshops, with information on each speaker, as well as all events and conference details.
 
May 21, 2013   VALIANT Clinical Trial of Ambulatory Type III Spinal Muscular Atrophy Patients Conducted at Ohio State University Published in Muscle and Nerve.
 
May 7, 2013   OKI Chapter Reaches Milestone 10th Annual Walk-n-Roll for Families of SMA
 
May 6, 2013   Annual SMA Conference Registration Deadline Now Only Three Days Away.
Registration has been filling quickly with this record breaking West Coast Conference.
 
May 2, 2013   Families of SMA Awards $150,000 to Dr. Burghes and Dr. Lorson to Investigate New Antisense Therapies for Spinal Muscular Atrophy.
 
May 1, 2013   Milestone Events Reaching New Heights for Families of Spinal Muscular Atrophy
This spring some of our key events are celebrating their 10th year in support of funding a treatment and a cure for SMA.
 
April 30, 2013   Families of SMA 2013 Update on the Spinal Muscular Atrophy Drug Pipeline.
15 new drugs now in development for SMA. 3 in active clinical trials. 11 companies investing in SMA drug programs.
 
April 26, 2013   2013 Walk n Roll to Cure SMA Update from Families of Spinal Muscular Atrophy.
Over 4,500 participants already registered.
 
April 23, 2013   ISIS Pharmaceuticals Initiates Clinical Study in Infants with Spinal Muscular Atrophy.
 
April 22, 2013   Families of SMA is Thrilled to announce that Registration for The 2013 Annual SMA Conference Has Now Exceeded over 1200 People.
With less then 4 weeks remaining until registration closes, the 2013 Annual SMA Conference, held on June 13th – June 16th, will be a record breaking West Coast conference for Families of SMA.
 
April 16, 2013   Families of Spinal Muscular Atrophy Announces Panelists for The Research Q and A at The 2013 Annual SMA Conference.
The Annual SMA Conference will bring together 1300 families and researchers from around the world. A Research Q&A Session will close the conference, where leading experts will address new advances, strategies, and challenges in SMA drug development.
 
April 13, 2013   Families of SMA Legislative Update: NIH Describes Spinal Muscular Atrophy Related Research Efforts to Congress
 
April 12, 2013   Agenda for The 2013 Annual Spinal Muscular Atrophy Conference is Now Available Online - 40 SMA Workshop Topics
Families of SMA is excited to announce the agenda, which includes times and workshop titles for The 2013 Annual SMA Conference at Disneyland in Anaheim, California, from June 13th – June 16th.
 
April 11, 2013   Families of Spinal Muscular Atrophy Offers Valuable Scholarship Opportunity for the Continuing Medical Education SMA Conference
Families of SMA is offering a Continuing Medical Education Conference of Interdisciplinary Perspectives on Spinal Muscular Atrophy: Defining Your Role, for medical professionals on Wednesday, June 12, 2013 prior to the start of the Annual SMA Conference and Researcher Conference in Disneyland-Anaheim, California.
 
April 10, 2013   Families of Spinal Muscular Atrophy Introduces New Family and Researcher Event to The 2013 Annual SMA Conference.
This Family and Researcher Poster Session and Reception is an exciting new addition to this year’s conference as it will allow families and researchers a wonderful opportunity to connect and learn from each other.
 
April 9, 2013   Time to Walk-n-Roll to Cure SMA
This spring, Walk-n-Rolls across the country will be helping to raise critical funds to advance a treatment and a cure for Spinal Muscular Atrophy.
 
April 8, 2013   Project Cure SMA Publishes Study on Infant Motor Scale for Use in Clinical Trials for Spinal Muscular Atrophy.
Tests to determine if new therapies are working are critical. Project Cure SMA recently published on the reliability and validity of the TIMPSI Test for Infants with SMA Type I. This measure will be further evaluated in the NIH NeuroNext SMA Biomarker Trial.
 
April 5, 2013   Over 250 Registered for The Annual SMA Conference through Newly Diagnosed Conference Scholarships Provided by Families of Spinal Muscular Atrophy.
Families of Spinal Muscular Atrophy is proud to again offer an amazing support program for all newly diagnosed SMA families to attend this year’s Annual SMA Conference in Anaheim, CA, June 13th – 16th, at the Disneyland Hotel.
 
April 4, 2013   Families of SMA Announces Grant to Cytokinetics for Preclinical Development of Tirasemtiv for Spinal Muscular Atrophy.
This award will support testing of Tirasemtiv in mouse models of Spinal Muscular Atrophy. It is the second of three drug discovery awards to be made by FSMA in recent months.
 
April 3, 2013   110 Research Presentations at The 17th Spinal Muscular Atrophy Research Group Meeting Hosted and Supported by Families of SMA.
An important way Families of SMA advances research is through The Annual SMA Research Group Meeting.
 
March 28, 2013   Families of Spinal Muscular Atrophy Thanks our Amazing Exhibitors for the 2013 Annual SMA Conference in Anaheim, CA.
 
March 20, 2013   Spinal Muscular Atrophy Clinical Trial Data Presented at the 2013 American Academy of Neurology (AAN) Meeting.
At the 65th Academy of Neurology (AAN) Meeting this week, data from Isis Pharmaceuticals on their Single Dose Phase I Clinical Trial for ISIS-SMNRx and from Project Cure SMA on their Carnival Type I Clinical Trial are being presented.
 
March 19, 2013   Families of SMA Launches New Program to Fund Care Research to Drive Improvements in Patient Care in Spinal Muscular Atrophy
 
March 13, 2013   Families of SMA and Nationwide Children’s Announce Multi-Million Dollar Award from NINDS to Advance CNS Gene Therapy for Spinal Muscular Atrophy
 
March 11, 2013   Meet Your Fundraising Goals with These Resources and Tips from Families of SMA
 
February 28, 2013   Trophos Announces Positive Interim Safety Data in Pivotal Study of Olesoxime in Spinal Muscular Atrophy
The Data Monitoring Committee (DMC) has reviewed the study data at one year and recommended to continue the study as planned through year two based on safety data and predefined stopping criteria.
 
February 28, 2013   Today is Rare Disease Day: Protect NIH Research Funding from Sequestration by Contacting Your Congressman.
Today people around the world are observing Rare Disease Day.
 
February 27, 2013   Families of SMA Spring Fundraising Events Season is in Full Swing to Fund Spinal Muscular Atrophy Research.
60 events from New York to California will raise over $1.6 Million to fund advancements in SMA research and critical family support and patient care programs.
 
February 26, 2013   Families of Spinal Muscular Atrophy Announces the Schedule for The 2013 Annual SMA Conference.
Preliminary details and schedule have now been announced for the incredibly magical 2013 Annual SMA Conference held June 13th – 16th in Anaheim, CA at the Disneyland Resort.
 
February 5, 2013   Families of Spinal Muscular Atrophy Offers a Medical Professional Conference at the 2013 Annual SMA Conference in Anaheim CA.
 
January 25, 2013   Recruitment Video Released for the NINDS NeuroNEXT SMA Biomarker Trial
The trial is currently enrolling infants with SMA, ages 0 to 6 months, at 15 different locations in the US.
 
January 18, 2013   Families of Spinal Muscular Atrophy Awards $150,000 to Dr. Lee Rubin at Harvard to Advance a New SMA Drug Discovery Program.
This is the first of three drug discovery awards to be made by Families of SMA in the next few months.
 
January 15, 2013   Families of Spinal Muscular Atrophy Advocacy Update 2013
Families of SMA’s Legislative Strategy is Leading the Way Towards the Development of New SMA Therapies.
 
January 15, 2013   Clinical Project Opportunities for SMA Patients and Families.
Click here for more information about several research projects that are currently enrolling.
 
January 11, 2013   New Edition of the Families of Spinal Muscular Atrophy Directions Newsletter Now Available.
2013 Conference Special.
 
January 10, 2013   Families of SMA Collaborating with Parent Project Muscular Dystrophy on Perceptions in Neuromuscular Community on Therapeutics and Clinical Trials
Has your child participated in a clinical trial (a trial testing a potential treatment) within the past 5 years? Click here to find out how to participate.
 
January 9, 2013   Annual Campaign for Families of Spinal Muscular Atrophy Exceeds Half Million Goal
Over half a million dollars raised in one month for SMA Research and Patient Support. Goal now raised to $750,000!
 
January 8, 2013   Families of Spinal Muscular Atrophy Receives Generous Funding from Corey and Erin Quinnell for Chilly Pads in the SMA Care Package Program.
 
January 3, 2013   Pfizer Licenses Families of Spinal Muscular Atrophy Quinazoline Drug Program from Repligen
Pfizer to Develop Families of SMA Quinazoline Drug Program.
 
December 31, 2012   Families of Spinal Muscular Atrophy Committed $2.5 Million to Funding SMA Research in 2012.
 
December 27, 2012   Families of Spinal Muscular Atrophy Awards $710,000 for Basic Research to Identify New and More Effective Approaches for SMA Therapies.
 
December 23, 2012   Families of Spinal Muscular Atrophy Awards New Research Funding of $140,000 to Dr. Christine DiDonato at Northwestern University.
Over the next several months, Families of SMA will award over $1 Million in new research funding.
 
December 21, 2012   Families of Spinal Muscular Atrophy Awards New Research Funding of $140,000 to Dr. Custer in the Androphy Lab at Indiana University
Over the next several months, Families of SMA will award over $1 Million in new research funding.
 
December 20, 2012   NINDS Releases Update on the SMA Project.
Click here to read about the NINDS Therapeutic Project for Spinal Muscular Atrophy.
 
December 19, 2012   Families of Spinal Muscular Atrophy Awards New Research Funding of $140,000 to Dr. Jocelyn Cote at the University of Ottawa.
Over the next several months, Families of SMA will award over $1 Million in new research funding.
 
December 17, 2012   Families of Spinal Muscular Atrophy Awards New Research Funding of $75,000 to Dr. Chien-Ping Ko at the University of Southern California
Over the next several months, Families of SMA will award over $1 Million in new research funding.
 
December 14, 2012   Families of Spinal Muscular Atrophy Awards New Research Funding of $75,000 to Dr. Yong-Chao Ma at Northwestern University.
Over the next several months, Families of SMA will award over $1 Million in new research funding.
 
December 13, 2012   Annual Campaign for Families of Spinal Muscular Atrophy Reaches Half Way Goal with $250,000 to Fund SMA Research and Patient Support.
Advance Research. Support Families. GIVE Hope.
 
December 12, 2012   Families of Spinal Muscular Atrophy Awards New Research Funding of $140,000 to Dr. Charlotte Sumner at Johns Hopkins University.
Over the next several months, Families of SMA will award over $1 Million in new research funding.
 
December 11, 2012   The Families of Spinal Muscular Atrophy Medical Advisory Council Meets to Advance SMA Patient Care and Family Support
 
December 4, 2012   NIH's RAC Approves Infant Trial for Systemic AAV9-SMN Gene Therapy for SMA. FDA Approval to Be Sought in 2013.
The Kaspar team at Nationwide Children's Hospital presented their program to the NIH Recombinant Advisory Committee (RAC) today. In the coming months, the team will submit an IND to the FDA for final approval to begin human clinical trials. FSMA funding is helping move this program into older and bigger patients with SMA.
 
December 3, 2012   The Miller McNeil Woodruff Foundation Donates $87,000 to the 2013 Annual Campaign of Families of SMA.
$1,000 for every day sweet Miller was here with us!
 
November 26, 2012   Families of SMA Announces #GivingTuesday Social Media Campaign
GIVE Hope on Giving Tuesday!
 
November 21, 2012   Families of Spinal Muscular Atrophy Launches Annual Campaign to Support SMA Research and Patient Support.
Advance Research. Support Families. Give Hope.
 
November 20, 2012   The Head Start Early Registration Deadline for The 2013 Annual SMA Conference Approaches.
This Head Start Early Registration Fee will be valid through December 2nd 2012, at 11:59pm Central Standard Time, when registration prices will increase.
 
November 9, 2012   Over One Hundred Events Bring Together 19,500 Participants to Raise Over $1.45 Million for Spinal Muscular Atrophy Research and Support.
Thank you to our amazing Chapter Leaders and SMA families who spend countless hours planning these wonderful fundraising events in support of finding a treatment and a cure for Spinal Muscular Atrophy.
 
November 8, 2012   Nature Medicine Publishes Article on Targeted Therapies for SMA
The article titled "Call in the Backup" focuses on novel therapies for SMA and the companies involved.
 
November 7, 2012   The Deadline to Apply for a Conference Scholarship for the 2013 Annual SMA Conference Approaches.
Through generous donations, fundraisers and sponsorships, SMA families can apply for a chance to receive a registration scholarship to attend the 2013 Annual SMA Conference held at Disneyland, CA.
 
November 7, 2012   FSMA Translational Advisory Committee and Scientific Advisory Board Meet to Evaluate New Spinal Muscular Atrophy Research Funding.
Last week the SAB met to assess 37 basic research grant applications for funding. The TAC met to evaluate 7 drug discovery projects for SMA.
 
November 5, 2012   Families of Spinal Muscular Atrophy Offers Help to SMA Families Affected by Hurricane Sandy.
Our thoughts are with all those families on the East Coast who have been affected by Hurricane Sandy.
 
November 5, 2012   Families of SMA Nominates Spinal Muscular Atrophy as a Disease for FDA’s Patient-Focused Drug Development Initiative
Families of SMA has submitted formal comments to the FDA urging the agency to include SMA on the list of diseases that will receive a public meeting as part of this initiative. The comments make clear to the FDA that the severity of SMA and the tremendous progress made towards developing a robust therapy pipeline positions SMA as a perfect candidate to benefit from the new Patient-Focused Drug Development program.
 
October 26, 2012   Families of SMA Alabama Chapter Walk-n-Roll to Cure Spinal Muscular Atrophy Reaches Milestone 10th Annual Year.
The 10th Annual Alabama Chapter Walk-n-Roll to Cure SMA, held October 6th at Snow Hinton Park in Tuscaloosa, AL was a tremendous success!
 
October 24, 2012   Isis Initiates Safety, Tolerability and Dose-range Finding Study of Multiple Doses in Patients with Spinal Muscular Atrophy
This is an open-Label, dose escalation study to assess the safety, tolerability and dose-range finding of multiple doses of the drug delivered intrathecally to 24 patients with SMA. It follows up on a single dose safety study completed earlier this year.
 
October 23, 2012   Check Out Families of SMA’s New Event Materials
SMA families will stand above the rest at their next event thanks to these exciting new display items.
 
October 18, 2012   Families of Spinal Muscular Atrophy Awards Funding for Patient Travel in The SMA Biomarker Trial Conducted by New NINDS Clinical Trial Network.
 
October 17, 2012   Families of SMA Publishes a New and Comprehensive SMA Care Series Booklet Called Understanding Spinal Muscular Atrophy.
This new SMA Care Series Booklet gives an in depth overview of Spinal Muscular Atrophy written for SMA families and Medical Care Professionals.
 
October 15, 2012   Spinal Muscular Atrophy Researchers Participate at The 2012 Society of Neuroscience Meeting.
The Society of Neuroscience Meeting is the premiere neurobiology meeting annually with over 30,000 scientists attending. This year multiple presentations on Spinal Muscular Atrophy will be given by scientists funded by Families of SMA.
 
October 10, 2012   Spinal Muscular Atrophy Studies Conducted at Columbia University Published in Prestigious Journal Cell.
Two studies by scientists in the McCabe, Pellizzoni, and Mentis laboratories at the Motor Neuron Center at Columbia University Medical Center (CUMC) suggest that dysfunction in motor circuits is critical in Spinal Muscular Atrophy. Families of SMA provided funding to the Mentis laboratory for this work.
 
October 3, 2012   Families of Spinal Muscular Atrophy Receives Funding from The Charles Rosenbaum Foundation for EasyS Medical Strollers for the FSMA Equipment Pool.
 
October 2, 2012   Families of SMA Releases Family Support and Patient Services Guide as the Latest Spinal Muscular Atrophy Care Series Booklet.
 
September 28, 2012   The Spinal Muscular Atrophy Community Grieves the Loss of Families of SMA Board Member and Community Leader Amy Marquez.
Everyone at Families of SMA sends our condolences to the family of Amy Marquez.
 
September 26, 2012   Repligen Initiates Phase 1b Trial of RG3039 for Spinal Muscular Atrophy Licensed from Families of SMA.
The primary objectives of the study are to further evaluate the safety and plasma pharmacokinetics (PK) of multiple doses of RG3039 in healthy volunteers. Repligen licensed RG3039 in 2009 from Families of Spinal Muscular Atrophy (FSMA).
 
September 25, 2012   Families of Spinal Muscular Atrophy Receives Generous Funding from John and Lacey Beeson to fund Z-Flo Positioners for the SMA Care Package Program.
Families of SMA is incredibly grateful to receive generous funding from John and Lacey Beeson for the Newly Diagnosed SMA Care Package Program to fund Z-Flo™ Fluidized Positioners to include in every Type I Care Package, in memory of their son, Hayden Riley Beeson.
 
September 24, 2012   10th Annual Northern California Walk-n-Roll For Families of SMA Raises Record Total in Milestone Year.
The 10th Annual Northern California Walk-n-Roll, held August 25 at Golden Gate Park in San Francisco, CA, was a tremendous success!
 
September 21, 2012   Registration for the 2013 Annual SMA Conference, held in Anaheim, California June 13th – June 16th, at The Disneyland Hotel, is now available.
We are now offering the Head Start Early Registration Fee which will be valid through December 1st, 2012, at 11:59pm Central Standard Time, when registration prices will increase.
 
September 20, 2012   Funding Received from Sweet Baby Zane and Hillary & Keith Schmid Has Reached Amazing Levels Enabling More SMA Families to Receive Necessary Equipment.
Families of SMA is thrilled to receive additional generous funding from Keith & Hillary Schmid and Sweet Baby Zane to purchase necessary medical equipment for SMA families through the FSMA Equipment Pool.
 
September 19, 2012   Families of SMA Receives Seven Full Proposals for Funding New Drug Discovery Projects for Spinal Muscular Atrophy.
They will be assessed by our Translational Advisory Committee (TAC) in November of 2012.
 
September 18, 2012   Videos and Presentations from the Families of SMA Continuing Medical Education Conference for Spinal Muscular Atrophy Now Available.
The videos and presentations from the Continuing Medical Education Conference for medical professionals, titled “Interdisciplinary Perspectives on Spinal Muscular Atrophy: Defining Your Role”, are now available on the FSMA website.
 
September 17, 2012   SMA Families Across the Country Make Waves During Spinal Muscular Atrophy Awareness Month.
Throughout the month of August, the SMA Community joined together to raise awareness of Spinal Muscular Atrophy and hosted dozens of awareness events across the country!
 
September 13, 2012   NINDS Launches First Trial, A Biomarker Study for Spinal Muscular Atrophy, with Innovative, New National Clinical Trial Network.
The Network for Excellence in Neuroscience Clinical Trials (NeuroNext) is a twenty-five site national clinical trial network created by NINDS of the NIH to test promising new therapies for both pediatric and adult patients with neurological diseases. The first study to be conducted by NeuroNext is a Biomaker and Natural History Study in infants with Type I SMA. FSMA will be collaborating with NeuroNext and funding patient travel for the study.
 
September 7, 2012   Families of Spinal Muscular Atrophy Receives 36 Basic Research Grant Applications for New Funding.
These research applications received from around the world will be assessed by our Scientific Advisory Board this fall.
 
September 6, 2012   Imminent Spending Cuts Threaten to Curtail Expanding Federal Investment in SMA Research.
Several New NIH-Sponsored SMA Initiatives are in Jeopardy if Congress Fails to Prevent Sequestration.
 
September 4, 2012   The 2012 Annual Spinal Muscular Atrophy Conference in MN was the Most Successful Midwest Conference Yet.
The final attendance number was almost 1,200, which is a wonderful showing of families, researchers and professionals looking to connect with each other in the SMA Community.
 
August 21, 2012   Families of Spinal Muscular Atrophy Walk-n-Rolls are Coming to a City Near You.
This fall, SMA families from around the country will be hosting 23 Walk-n-Rolls helping to raise critical funds to push the advancement of a treatment and a cure for Spinal Muscular Atrophy.
 
August 20, 2012   Photos Posted for Annual SMA Candle Lighting for Spinal Muscular Atrophy Awareness Month.
Many photos taken of children and families during the Annual SMA Candle Lighting are now posted on the Families of SMA Facebook Page.
 
August 14, 2012   Let’s Play Ball…Join Families of SMA at the Ballparks to Raise Awareness for Spinal Muscular Atrophy.
Several Major League Baseball teams are teaming up with Families of SMA to help raise awareness and funds for finding a treatment and a cure for Spinal Muscular Atrophy
 
July 19, 2012   The 2012 Annual Spinal Muscular Atrophy Conference held in Bloomington, MN was a Tremendous Success.
 
July 18, 2012   Families of Spinal Muscular Atrophy Announces Four Winners of The 2012 Annual SMA Conference iPad Survey Giveaway.
 
July 17, 2012   Families of Spinal Muscular Atrophy Releases Summary of the Researcher Question and Answer Session at The 2012 Annual SMA Conference.
The 2012 Annual SMA Conference brought together 1,200 families and researchers from around the world to Minnesota this June.
 
July 16, 2012   University of Wisconsin Research Survey Study for Families and Individuals with Spinal Muscular Atrophy.
 
July 16, 2012   Highlights from The 16th Annual Spinal Muscular Atrophy Research Group Meeting.
Two hundred researchers attended the conference, representing over fifty academic organizations and thirteen companies, from thirteen countries around the world.
 
July 13, 2012   Submitted Photos from the 2012 Annual Spinal Muscular Atrophy Conference Now Posted on the FSMA Facebook Page.
Many photos taken of children, families and researchers at the 2012 Annual SMA Conference in Bloomington, MN are posted on the Families of SMA Facebook Page.
 
July 12, 2012   SMA Home for the Cure Completed to Support Families of Spinal Muscular Atrophy.
 
July 11, 2012   President Obama Signs Into Law a Bill to Expedite FDA Approval of Drugs for Rare Diseases.
 
July 5, 2012   Families of SMA Releases a Request for Proposals for Basic Research Projects in Spinal Muscular Atrophy.
Families of SMA is pleased to announce that we are accepting grant applications for 2012 funding of basic research projects, under a competitive review by our Scientific Advisory Board (SAB).
 
June 28, 2012   Families of SMA Releases Request for Proposals for Preclinical Drug Projects in Spinal Muscular Atrophy.
FSMA invites proposals for pre-clinical drug discovery programs for Spinal Muscular Atrophy (SMA), under a competitive review by our Translational Advisory Committee (TAC).
 
June 15, 2012   Agenda and Conference Booklet for The 2012 Annual Spinal Muscular Atrophy Conference Now Available Online.
This 76 page booklet includes the detailed agenda of all conference workshops, including information on each conference speaker and all conference events.
 
June 15, 2012   U.S. Senate Appropriations Committee Approves Report Language Requested by Families of SMA.
Language Urges NIH to Continue Efforts that Support Development of Spinal Muscular Atrophy Drug Therapies and SMA Standards of Care.
 
June 13, 2012   FSMA Chapters and Families Raise Over $4.6 Million in the Past Year to Support Spinal Muscular Atrophy Research and Support Programs.
Hundreds of fundraising events help Families of SMA to fund critical research and family support programs.
 
June 12, 2012   Congress Close to Passing Bill to Expedite FDA Approval of Drugs for Rare Diseases
 
June 7, 2012   Families of SMA Announces $1.4 Million Planned for Next Round of Spinal Muscular Atrophy Research Funding.
Families of Spinal Muscular Atrophy (FSMA) is dedicated to creating a treatment and cure for Spinal Muscular Atrophy (SMA) by funding and advancing a comprehensive research program.
 
June 6, 2012   New Edition of Families of Spinal Muscular Atrophy Community Newsletter Directions Now Available.
This Newsletter is one way we try and build our SMA community and bring us all closer together by sharing stories and experiences.
 
June 1, 2012   Families of SMA Awarded Grant from the National Institute of Health for The Annual Spinal Muscular Atrophy Research Group Meeting.
We thank NINDS at the NIH for the fantastic support that helps bring together the SMA research community along with our families.
 
May 23, 2012   Families of Spinal Muscular Atrophy Announces Sponsors for the Upcoming Annual SMA Conference.
Families of SMA is pleased to announce that we currently have 24 Sponsors for the upcoming 2012 Annual SMA Conference.
 
May 17, 2012   Families of Spinal Muscular Atrophy Announces the 2012 Annual SMA Conference Workshops.
 
May 15, 2012   Families of Spinal Muscular Atrophy Receives Additional Shipment of Wagons from Radio Flyer in Support of the SMA Care Package Program.
Radio Flyer, Inc. donates an additional 100 Pathfinder wagons to support the mission of Families of SMA.
 
May 10, 2012   Families of Spinal Muscular Atrophy Announces $700,000 Funding for Novel Drug Program at Newly Created California Institute for Biomedical Research
 
May 7, 2012   Families of Spinal Muscular Atrophy Announces Keynote Speakers and Research Q&A Participants at The 2012 Annual SMA Conference.
 
May 4, 2012   Families of Spinal Muscular Atrophy Releases The Nutrition Basics Care Series Booklet Translated into Spanish.
Families of SMA has a new program of translating all of the SMA Care Series Booklets into Spanish editions.
 
May 2, 2012   Families of SMA Awards New Funding to Advance a CNS Delivered Gene Therapy for Spinal Muscular Atrophy
 
April 30, 2012   Families of Spinal Muscular Atrophy Thanks Sweet Baby Zane for Providing Car Beds and Ez-On Vests
The Families of SMA Equipment Pool recently received an additional 14 Car Beds and 30 E-Z-On Vests to be sent to newly diagnosed SMA Type I families.
 
April 27, 2012   Concert for a Cure Targets Million Dollar Milestone Supporting Spinal Muscular Atrophy Research
 
April 25, 2012   Repligen Reports Positive Results From Phase 1 Clinical Trial of RG3039 for Spinal Muscular Atrophy.
 
April 24, 2012   SMA Home for the Cure Kick-off and Frame-up to Support Families of Spinal Muscular Atrophy.
House to be Built and Sold Entirely to Support Families of SMA Research and Support Programs.
 
April 23, 2012   Abstracts Presented on Spinal Muscular Atrophy at the 2012 American Academy of Neurology (AAN) Meeting.
SMA abstracts will be presented to 10,000 clinicians and researchers in attendance.
 
April 17, 2012   Research Presentations Announced for the 16th Spinal Muscular Atrophy Research Group Meeting Hosted and Supported by Families of SMA.
An important way Families of SMA advances Spinal Muscular Atrophy research is The Annual SMA Research Group Meeting.
 
April 13, 2012   Families of SMA Thanks our 31 Amazing Vendors for the upcoming 2012 Annual SMA Conference in Bloomington, MN.
 
March 27, 2012   Families of Spinal Muscular Atrophy Receives Generous Funding to purchase Special Bath Chairs for the FSMA Equipment Pool.
Families of SMA is incredibly grateful to receive funding from a generous California donor to purchase Leckey Advance Bath Chairs.
 
March 22, 2012   Be a Part of Something Big...Sign Up and Participate in a Families of Spinal Muscular Atrophy Spring Fundraiser
Families of SMA will be hosting over 60 fundraising events across the country this spring, aiming to raise over $2 Million for SMA research and support programs.
 
March 20, 2012   Jacob Isaac Rappoport Foundation Aims for Million Dollar Milestone Supporting SMA Research
 
March 19, 2012   Families of Spinal Muscular Atrophy Introduces a New Component for Medical Professional Education at the 2012 Annual SMA Conference.
Families of SMA is offering a Continuing Medical Education Conference titled “Interdisciplinary Perspectives on Spinal Muscular Atrophy: Defining Your Role”, for medical professionals.
 
March 15, 2012   Families of SMA Funded Study in the Journal of Neuroscience Shows Restoring SMN in Motor Neurons Corrects Motor Defects in Mice.
Dr. Christine DiDonato and colleagues at Northwestern University publish a paper on enhancing SMN specifically in motor neurons in the Journal of Neuroscience.
 
March 12, 2012   Families of Spinal Muscular Atrophy Adds Dr. Elliot J. Androphy to Scientific Advisory Board.
 
March 5, 2012   Families of Spinal Muscular Atrophy Receives a Record Number of Vendors for the upcoming 2012 Annual SMA Conference.
Families of SMA is thrilled to announce that we have just received our 21st Vendor for the 2012 Annual SMA Conference.
 
February 24, 2012   NIH Provides Congress an Overview of the Federal Government’s Spinal Muscular Atrophy Research Activities.
NIH also releases 2011 funding amounts for Spinal Muscular Atrophy Research.
 
February 22, 2012   Families of SMA Funded Researchers Publish Review Article on Role of SMN in Motor Axons
Drs. Claudia Fallini, Gary Bassell, and Wilfried Rossoll of Emory University publish paper in Brain Research.
 
February 17, 2012   American Medical Association Publishes Article on NINDS Clinical Trial Network NeuroNEXT with First Study on Spinal Muscular Atrophy.
Clinical Trial Network Removes Barriers Common to Studies of Neurological Diseases. First Study to be on SMA Biomarkers.
 
February 15, 2012   Families of Spinal Muscular Atrophy Releases The Breathing Basics Care Series Booklet Translated into Spanish.
Families of SMA has begun a new program of translating all of the SMA Care Series Booklets into Spanish editions. These booklets will be on hand at the FSMA National Office as well as available for download on the Families of SMA website at www.CureSMA.org.
 
February 10, 2012   Annual Campaign for Families of Spinal Muscular Atrophy Exceeds $500,000 for Funding New SMA Research.
 
February 7, 2012   Families of Spinal Muscular Atrophy Receives 250 Baby Einstein Discovery Kits for the Newly Diagnosed SMA Care Package Program.
Families of SMA is so incredibly grateful to receive a donation of 250 Baby Einstein Discovery Kits, which is one of the items now included in all Newly Diagnosed Care Packages, from Paula Lavigne and Chris Arnold in memory of their son, Wyatt Teagan Arnold.
 
January 30, 2012   Families of Spinal Muscular Atrophy Receives $25,000 Sponsorship from BAYADA Home Health Care for The 2012 Annual SMA Conference.
Families of Spinal Muscular Atrophy is excited to announce a significant sponsorship for The 2012 Annual SMA Conference.
 
January 26, 2012   New Winter Edition of the Families of Spinal Muscular Atrophy Directions Newsletter is Now Available Online.
The Winter 2011 edition of the family support and SMA community newsletter Directions is now available.
 
January 25, 2012   Deadline for Early Registration for the Annual Spinal Muscular Atrophy Conference Approaches.
This head start early registration fee will be valid through February 1st, 2012, at 11:59pm Central Standard Time, when registration prices will increase.
 
January 19, 2012   Families of Spinal Muscular Atrophy Welcomes the New Carolinas Chapter and Satellite Chapter Capital Region of Greater New York.
 
January 18, 2012   Update on the Spinal Muscular Atrophy Drug Development Pipeline from Families of SMA.
 
January 13, 2012   Families of SMA Receives Funding for Bath Pads for Care Packages from The Joseph Lillo Spinal Muscular Atrophy Foundation.
Families of SMA is incredibly grateful to receive funding to purchase a 150 of the Leacho Safer Bather Infant Bath Pads, which are one of the items in all Newly Diagnosed Type I Care Packages.
 
January 12, 2012   Families of SMA is Proud to Have Contributed Funding to Key Spinal Muscular Atrophy Research Results Published in 2011.
Funding for this work was awarded to 14 institutions resulting in 13 research articles published with FSMA support in 2011.
 
January 10, 2012   Families of Spinal Muscular Atrophy Releases SMA Care Series Booklet on Nutrition Basics for SMA.
“Nutrition Basics: Fostering Health and Growth for Spinal Muscular Atrophy” is Families of SMA’s newest release of the SMA Care Series Booklets, which are provided to all newly diagnosed families, as well as SMA Care Providers worldwide.
 
January 4, 2012   Biogen Idec and Isis Pharmaceuticals Announce Global Collaboration for Antisense Program Targeting Spinal Muscular Atrophy.
 
December 29, 2011   Families of Spinal Muscular Atrophy Raises Annual Campaign Goal to $500,000 to Support New SMA Research.
Help fund new SMA research to expand the drug pipeline by making your year-end gift to Families of SMA today!
 
December 27, 2011   Families of Spinal Muscular Atrophy Releases The Genetics of SMA Care Series Booklet Translated into Spanish.
Families of SMA has begun a new program of translating all of the SMA Care Series Booklets into Spanish editions.
 
December 22, 2011   Single Administration of Antisense Oligomer Delivered to the CNS Rescues a Severe Mouse Model of Spinal Muscular Atrophy.
The Burghes laboratory at Ohio State University publishes a paper in Human Molecular Genetics showing a single dose of an antisense oligomer (ASO) can greatly benefit survival, weight gain, and motor function in a severe mouse model of SMA.
 
December 20, 2011   Families of Spinal Muscular Atrophy is Thrilled to Receive Additional Funding for the Type I SMA Care Packages.
The Jacob Isaac Rappoport Foundation and Adi & Shaina Rappoport have awarded Families of SMA with another year of funding for the Type I Care Packages, sent to all newly diagnosed Type I families as soon as they contact Families of SMA.
 
December 19, 2011   Isis Pharmaceuticals Initiates Phase 1 Clinical Study in Patients with Spinal Muscular Atrophy.
Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study of ISIS-SMNRx in patients with spinal muscular atrophy (SMA). Isis is developing ISIS-SMNRx as a potential treatment for all types of SMA.
 
December 16, 2011   Families of Spinal Muscular Atrophy is Pleased to Announce that the Annual Campaign Has Raised Over $155,000 in Just Four Weeks.
Families of SMA has reached the half way point toward the fundraising goal for the 2012 annual campaign.
 
December 15, 2011   Families of Spinal Muscular Atrophy Announces Special Session Topic for the 16th Annual SMA Research Group Meeting.
An important way Families of SMA advances SMA research is the Annual SMA Research Group Meeting. We invest approximately $200,000 each year to bring researchers from around the world together.
 
December 14, 2011   Families of Spinal Muscular Atrophy Releases SMA Care Series Booklet Translated into Spanish, for Newly Diagnosed Type I Families.
Families of SMA has begun a new program of translating all of the SMA Care Series Booklets into Spanish editions.
 
December 12, 2011   Families of SMA Awards New Funding for The Spinal Muscular Atrophy Registry.
Families of SMA has awarded new funding of $60,000 to support The Spinal Muscular Atrophy Patient Registry over the next year.
 
December 8, 2011   Families of Spinal Muscular Atrophy Hits Annual Campaign Milestone to Benefit SMA Research and Support Programs.
Contributions from Annual Campaign top $100,000; one-third of the way to the $300,000 goal.
 
December 7, 2011   Unique Dinner and Gala Events Raise Funds and Awareness for Spinal Muscular Atrophy.
Families of SMA can help you plan a fun and innovative Dinner/Gala event.
 
December 6, 2011   Newly Funded Spinal Muscular Atrophy Researcher Profile at Families of SMA: Dr. Louise Simard.
Dr. Simard is Professor and Head of Biochemistry and Medical Genetics at the University of Manitoba and has been awarded a basic research grant for $25,000 from Families of SMA.
 
December 2, 2011   Families of Spinal Muscular Atrophy 2012 Annual Campaign Starts Off Strong Raising Funds for SMA Research.
First ten days of 2012 Annual Campaign raise over $60,000.
 
December 1, 2011   Hopkins Medical Magazine Showcases Cover Article on Spinal Muscular Atrophy Featuring Families of SMA Funded Johns Hopkins Researchers.
The Fall 2011 issue of this publication from Johns Hopkins focuses on the important work of two SMA researchers.
 
November 30, 2011   Families of Spinal Muscular Atrophy Announces Applications to Receive a 2012 Annual SMA Conference Registration Scholarship Now Open.
Families of Spinal Muscular Atrophy will once again be offering SMA families the chance to receive a financial need based conference registration scholarship to the 2012 Annual SMA Conference in Minneapolis, MN.
 
November 29, 2011   Roche Licenses Spinal Muscular Atrophy Compounds from PTC Therapeutics for Development.
Please click here to learn more about the Roche and PTC agreement.
 
November 28, 2011   Newly Funded Spinal Muscular Atrophy Researcher Profile at Families of SMA: Dr. Umrao Monani.
Dr. Monani is an Assistant Professor at Columbia University and has been awarded a basic research grant for $160,000 from Families of SMA.
 
November 22, 2011   Families of Spinal Muscular Atrophy Unveils Annual Campaign Goal To Fund New SMA Research and Support Initiatives.
The 2012 Annual Campaign for Families of SMA aims to raise over $300,000 to fund new basic and drug discovery research projects, family support and patient services programs.
 
November 21, 2011   Newly Funded Spinal Muscular Atrophy Researcher Profile at Families of SMA: Dr. Stephen Kolb
Dr. Kolb is an Assistant Professor of Neurology at The Ohio State University and has been awarded a basic research grant for $70,000 from Families of SMA.
 
November 16, 2011   Families of Spinal Muscular Atrophy Announces the Schedule for The 2012 Annual SMA Conference.
The schedule is announced for the 2012 Annual SMA Conference held June 21st – 24th in Bloomington, MN.
 
November 11, 2011   Newly Funded Spinal Muscular Atrophy Researcher Profile at Families of SMA: Drs. Rashmi Kothary and Lyndsay Murray
Drs. Kothary and Murray are researchers at Ottawa Hospital Research Institute and have been awarded a basic research grant for $120,000 from Families of SMA.
 
November 10, 2011   Families of Spinal Muscular Atrophy Can Help You Organize a Walk-n-Roll Event to Benefit SMA Research and Support Programs.
Hosting a Walk-n-Roll event made easy with materials and assistance from Families of SMA.
 
November 9, 2011   Families of Spinal Muscular Atrophy Funded Researchers Participate at the 2011 Society of Neuroscience Meeting
This year multiple presentations on Spinal Muscular Atrophy will be given by scientists funded by Families of SMA. Events at SfN will also included the annual SMA Satellite Symposium co-funded by FSMA.
 
November 8, 2011   SMA Community Connections, the Social Networking Site from Families of Spinal Muscular Atrophy.
SMA Community Connections at www.SMACommunity.org is an incredible support site for SMA families worldwide.
 
November 7, 2011   Newly Funded Spinal Muscular Atrophy Researcher Profile at Families of SMA: Dr. Christopher Henderson
Dr. Henderson is a Professor at Columbia University and has been awarded a basic research grant for $160,000 from Families of SMA.
 
November 2, 2011   Families of Spinal Muscular Atrophy Announces Details on Newly Diagnosed Conference Scholarship Program.
 
October 31, 2011   Newly Funded Spinal Muscular Atrophy Researcher Profile at Families of SMA: Dr. Wilfried Rossoll and Dr. Claudia Fallini.
Drs. Rossoll and Fallini are at Emory University and have been awarded a new basic research grant for $140,000.
 
October 28, 2011   Repligen Provides Update on the Phase I Trial for Spinal Muscular Atrophy Drug RG3039
The drug was licensed to Repligen for clinical development from Families of SMA in 2009. FSMA fully managed and funded this program to clinical development.
 
October 28, 2011   Two Families of Spinal Muscular Atrophy Research Award Recipients Lead New Neuromuscular Clinic for SMA.
Christine DiDonato, PhD and Kristin Krosschell, PT/MA along with Nancy Kuntz, MD from Children’s Memorial Hospital in Chicago, IL seek to improve the quality of life for children with SMA.
 
October 27, 2011   Families of SMA Releases Updated Family Guide to Spinal Muscular Atrophy Research.
FSMA has just released an updated this booklet, which covers the basics of Spinal Muscular Atrophy research for SMA families.
 
October 25, 2011   Newly Funded Spinal Muscular Atrophy Researcher Profile at Families of SMA: Dr. Chien Ping Ko
Dr. Ko is a Professor at the University of Southern California and has been awarded a new basic research grant for $70,000 from Families of SMA. Dr. Ko is presenting at the Society for Neuroscience SMA Symposia this November. Click here for more about his background and research.
 
October 18, 2011   Families of Spinal Muscular Atrophy Awards 7 New Basic Research Grants for $745,000 to Develop New Approaches for SMA Therapies.
Each project is described within our Fall 2011 issue of the Compass newsletter. Click here to learn more.
 
October 13, 2011   Charlotte Sumner and James Van Meerbeke at Johns Hopkins University Publish Article Reviewing the Spinal Muscular Atrophy Drug Pipeline.
Dr. Sumner is a member of the FSMA Translational Advisory Committee and a 2007/2008 grant recipient from FSMA.
 
October 7, 2011   Team Families of SMA Running to New Heights
Families of SMA Marathon Program celebrates success in 2011 and ready to grow in 2012.
 
October 6, 2011   Publication on Antisense Therapy Delivers Correction of Severe Spinal Muscular Atrophy in Mice.
 
October 4, 2011   It’s Not Too Late To Host a 2011 Event To Benefit Families of Spinal Muscular Atrophy Research and Support Programs.
Families of SMA is ready to assist you in planning a Fall fundraising event.
 
October 3, 2011   NeuroNEXT: Network for Excellence in Neuroscience Clinical Trials Sites Announced.
First trial will be a multi-site Spinal Muscular Atrophy Biomarker Study.
 
September 27, 2011   Families of Spinal Muscular Atrophy Releases Updated Care Series Booklet for Newly Diagnosed SMA Type I Families.
Families of SMA releases a new version of “Caring Choices: for Family of Infants Newly Diagnosed with SMA Type I”.
 
September 26, 2011   The Senate Appropriations Committee Approved its Version of The Fiscal Year 2012 Labor-HHS-Education Appropriations Bill.
It includes two pieces of the report language on SMA: one on NeuroNEXT and one on newborn screening.
 
September 22, 2011   Families of Spinal Muscular Atrophy Announces Spring and Summer Fundraising Event Results.
Hundreds of fundraising events result in over $1.5 million raised for Families of SMA.
 
September 14, 2011   Families of Spinal Muscular Atrophy Receives Additional Funding from Sweet Baby Zane and the Schmid Family to Purchase Critically Necessary Car Beds.
The Families of SMA Equipment Pool received an additional 27 Car Beds to be sent to newly diagnosed SMA Type I families.
 
September 13, 2011   Families of Spinal Muscular Atrophy is Excited to Announce that the Registration for the 2012 Annual SMA Conference in Minnesota is Now Available!
Registration for the 2012 Annual SMA Conference, held in Bloomington, MN June 21st - June 24th, at the DoubleTree Hotel Bloomington – Minneapolis South, is now available.
 
September 9, 2011   Families of Spinal Muscular Atrophy Receives Large Shipment of Donated Wagons from Radio Flyer to the SMA Care Package Program.
Families of SMA receives another shipment of 50 Pathfinder wagons, donated from Radio Flyer, Inc.
 
September 9, 2011   Lockwood and Hoffmann Families Win Apple iPads Donated to FSMA for Spinal Muscular Atrophy Awareness Month.
Families of SMA is excited to announce the winners of two brand new Apple iPads; the Lockwood Family of Cincinnati, Ohio and the Hoffmann Family of Milwaukee, Wisconsin.
 
September 9, 2011   FDA Proposes to Revamp, Improve its Program for Approving Drugs for Rare Diseases
 
September 8, 2011   Trophos Completes Patient Enrollment in Pivotal Efficacy Study of Olesoxime in Spinal Muscular Atrophy
Over 160 patients have been recruited into the trial since its initiation in October 2010.
 
September 7, 2011   Article in Bloomberg Markets Describes Several Spinal Muscular Atrophy Drug Programs
The article mentions programs at Novartis, PTC Therapeutics, ISIS, and Repligen Corporation.
 
August 24, 2011   Families of SMA Encourages Promotion of SMA Awareness Month in August with iPad Giveaway.
One week remains for SMA Awareness Month in August. Families of Spinal Muscular Atrophy to award two individuals with a donated iPad based on media placements.
 
August 17, 2011   The UW Pediatric Pulmonary Center Hosted Another Successful Family Education Day for Spinal Muscular Atrophy
The Family Education Day for SMA was created by Dr. Mary Schroth, the Chair of the FSMA Medical Advisory Council, and held this past Saturday in Madison, WI.
 
August 5, 2011   Families of Spinal Muscular Atrophy is Thrilled to Announce The 2012 Annual SMA Conference, to be Held in Minneapolis, MN.
The 2012 Annual SMA Conference will be held from June 21st – June 24.
 
August 4, 2011   Highlights from the 15th Annual Spinal Muscular Atrophy Research Group Meeting.
The International SMA Research Group Meeting is the biggest Spinal Muscular Atrophy (SMA) research conference in the world. 225 Researchers attended the conference, representing 70 total institutions, 14 biotech and pharmaceutical companies, and 11 countries worldwide.
 
August 4, 2011   Isis Pharmaceuticals Posts Update on ISIS-SMNRx Program for Spinal Muscular Atrophy
The Article Details New Data Presented on Isis' ISIS-SMNRx at the 2011 Annual Spinal Muscular Atrophy Conference hosted by FSMA.
 
August 3, 2011   The 2011 Annual Spinal Muscular Atrophy Conference Received Support from the Florida Delegation.
Families of SMA is proud to have received the support of 4 members of the FL Congressional Delegation.
 
August 2, 2011   Paper Published on Systemic Gene Delivery in Large Species with Funding from Families of Spinal Muscular Atrophy.
Researchers at Nationwide Children's Hospital and OSU publish a paper on gene therapy for SMA in the journal Molecular Therapy.
 
August 2, 2011   Families of SMA Announces Two Winners of The 2011 Annual Spinal Muscular Atropy Conference Apple IPad Survey Giveaway.
Two SMA Families have been awarded Apple IPad’s which were so generously donated to Families of SMA.
 
July 29, 2011   Photos Taken at the 2011 Annual Spinal Muscular Atrophy Conference Now Posted on the SMA Community Site.
 
July 28, 2011   2011 Annual Spinal Muscular Atrophy Conference Presentations and Workshops on Video Now Available Online.
 
July 26, 2011   Families of Spinal Muscular Atrophy Releases Videos of the Keynote Speakers and the Leading Researchers Participating in the Family Q &A Session.
From the 2011 SMA Conference.
 
July 25, 2011   Families of Spinal Muscular Atrophy Promotes SMA Awareness Month in August with iPad Drawing.
By promoting SMA Awareness Month throughout August, Families of Spinal Muscular Atrophy will award two families each a donated iPad.
 
July 21, 2011   The 2011 Annual Spinal Muscular Atrophy Conference in Orlando, FL was an Attendance Record and Huge Success.
This year’s SMA conference in Disney World was certainly our most magical and successful conference yet.
 
July 19, 2011   Families of SMA Awards $3 Million in New Spinal Muscular Atrophy Research Funding.
 
July 5, 2011   Results of CARNI-VAL Clinical Trial Published: A Twelve Month Prospective, Open-label Trial of VPA and L-carnitine for Spinal Muscular Atrophy.
The second set of results from the Families of SMA funded CARNI-VAL clinical trial in ambulatory children published in PLoS ONE.
 
June 28, 2011   Repligen Receives U.S. Fast Track Designation and European Orphan Medicinal Product Recommendation for Spinal Muscular Atrophy.
 
June 16, 2011   Record Number of Corporate and Foundation Sponsors Support The 2011 Annual SMA Conference.
 
June 13, 2011   225 Spinal Muscular Atrophy Researchers to Attend the 15th Annual SMA Research Group Meeting Hosted by FSMA.
 
June 12, 2011   Families of Spinal Muscular Atrophy Announces Expected Attendance for The 2011 Annual SMA Conference Over 1,500 With Participants from 23 Countries.
 
June 9, 2011   NINDS Announces Request for Applications for a SMA Clinical Research Study
This study will assess potential biomarkers for SMA in order to facilitate future drug development trials. The study will be done using the NeuroNEXT clinical trial infrastructure being established at NINDS. See within for more details.
 
June 8, 2011   FSMA Announces Keynote Speakers and the Leading Researchers Participating in the Family Sessions at The Annual SMA Conference.
 
May 19, 2011   Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Clinical Trial in SMA.
First ever clinical trial approved for SMA with a novel drug specifically designed to treat the disease.
 
May 6, 2011   National Institute of Child Health and Human Development at the NIH Funds a Pilot Study of Newborn Screening for Spinal Muscular Atrophy.
 
May 2, 2011   Spinal Muscular Atrophy Clinical Trial Results and Future SMA Clinical Research Strategy Discussed in Latest Issue of “Compass” Newsletter.
Click here to download a copy.
 
April 28, 2011   Spinal Muscular Atrophy Legislative Update for 2011.
Click here for an update on current activities and future goals.
 
April 15, 2011   A Dozen Abstracts on Spinal Muscular Atrophy Presented at the 2011 American Academy of Neurology (AAN) Meeting
These include presentations by Project Cure SMA on their clinical trial results, by the OSU group on gene therapy, and by Trophos on their ongoing SMA trial testing olesoxime.
 
April 11, 2011   Notice of Intent to Publish a Request for Applications for a Spinal Muscular Atrophy Biomarker Study and Convene a Related Workshop
The National Institute of Neurological Disorders and Stroke (NINDS) intends to promote a new initiative by publishing a Funding Opportunity Announcement to solicit applications for clinical research studies that will accelerate and augment the validation of biomarkers for Spinal Muscular Atrophy.
 
April 11, 2011   Families of Spinal Muscular Atrophy Translational Advisory Committee (TAC) and Scientific Advisory Board (SAB) Meet to Evaluate New Research Funding.
On April 7th, the Families of Spinal Muscular Atrophy SAB met to assess 34 basic research grant applications from around the world for funding. The FSMA TAC met the next day to evaluate 5 new drug discovery programs for Spinal Muscular Atrophy.
 
March 30, 2011   Families of Spinal Muscular Atrophy Announces Generous Donation from Radio Flyer to the SMA Care Package Program.
Radio Flyer, Inc. donates 100 Pathfinder wagons to support the mission of Families of SMA.
 
March 4, 2011   Scientists from Genzyme Corporation and ISIS Pharmaceuticals Publish Paper Showing Antisense Oligonucleoties (ASOs) Ameliorate Symptoms of Severe SMA
ASOs designed to correct splicing of the SMN2 gene improve symptoms in a severe mouse model of SMA when delivered directly to the CNS.
 
March 2, 2011   Families of Spinal Muscular Atrophy Receives 34 Basic Research Grants for Funding Consideration by our Scientific Advisory Board.
The best will be selected for potential funding by the members of our Scientific Advisory Board in a meeting in April.
 
February 25, 2011   Families of Spinal Muscular Atrophy Announces Enhancements to Fundraising Event Websites for 2011.
New features available on the Families of SMA website to support fundraising efforts.
 
February 23, 2011   NIH Funding for Spinal Muscular Atrophy Research Up 60% Since 2008.
Click here to see details and information on future funding.
 
February 22, 2011   Families of Spinal Muscular Atrophy was Proud to Host Another Successful Medical Advisory Council Meeting.
Members of the Medical Advisory Council attended two days of meetings at the Families of SMA National Office on January 28th and 29th.
 
February 18, 2011   Study with Funding from FSMA Shows a Muscle Specific Intervention Improves Phenotype in a Mouse Model of Spinal Muscular Atrophy
Dr. Sumner and colleagues at Johns Hopkins University publish a study in the journal Human Molecular Genetics showing improvement in survival but not motor function by increasing the levels of the muscle modulator Insulin-like growth factor 1 (IGF-1).
 
February 9, 2011   Audrey Lewis Young Investigator Award Winner Publishes Study in Neuron Implicating Sensory Synapse Defects in Mouse Model of Spinal Muscular Atrophy
Dr. George Mentis of Columbia University, the first recipient of the FSMA Audrey Lewis Young Investigator Award, publishes a study in the prestigious scientific journal Neuron implicating sensory synapse defects in mouse model of SMA.
 
February 8, 2011   Families of Spinal Muscular Atrophy Adds Three New Members to Scientific Advisory Board (SAB)
They include Rashmi Kothary, Ph.D. of the Ottawa Hospital Research Institute, Samuel Pfaff, Ph.D. of the Salk Institute of Biological Studies, and Mark Rich, M.D., Ph.D. of Write State University.
 
February 8, 2011   MotorGraft Clinical Trial for SMA Placed on FDA Clinical Hold.
In December, California Stem Cell, Inc. announced an investigational new drug (IND) application with the US Food and Drug Administration (FDA) for approval to commence a Phase I safety study for Spinal Muscular Atrophy (SMA) Type I. Recently, CSC received an official response from the FDA. Click here to see the details.
 
January 31, 2011   New Mouse Models of Spinal Muscular Atrophy Developed.
Dr. Christine DiDonato and colleagues at Children's Memorial Research Center in Chicago, IL and Ottawa Health Research Institute in Ottawa, ON recently published 2 new mouse models of SMA in the Journal PLoS One with funding from Families of SMA.
 
December 17, 2010   Families of Spinal Muscular Atrophy is Proud to be Implementing a New Support Program for Newly Diagnosed SMA Families.
Starting this month, we will be providing all newly diagnosed SMA families with this resource as soon as they contact FSMA.
 
December 1, 2010   California Stem Cell Inc. Files IND to Commence Phase I Clinical Trial in Spinal Muscular Atrophy
California Stem Cell, Inc. (CSC) and Families of Spinal Muscular Atrophy (FSMA) announced today that CSC has filed an investigational new drug (IND) application with the US Food and Drug Administration (FDA) for approval to commence a Phase I safety study on a jointly-developed stem cell-derived motor neuron transplantation therapy for Spinal Muscular Atrophy (SMA) Type I.
 
November 11, 2010   Families of Spinal Muscular Atrophy Launches Next Phase of Drug Development Efforts for SMA.
Families of SMA announces Request for Proposals (RFP) for new drug programs to develop therapies for Spinal Muscular Atrophy. FSMA is aiming to fund two new preclinical drug development programs in 2011, with more to follow.
 
November 9, 2010   NIH Holds Conference on Spinal Muscular Atrophy Drug Development.
Click here to see a summary report.
 
October 18, 2010   Trophos Initiates Pivotal Efficacy Study of Olesoxime in Spinal Muscular Atrophy
Trophos SA, a clinical stage pharmaceutical company announced the initiation of the pivotal efficacy study of olesoxime in SMA.
 
September 8, 2010   New Features Added to SMA Community Connections.
If you have not already joined SMA Community Connections, sign up today at www.smacommunity.org to check out our newest features!
 
August 24, 2010   Project Cure SMA Clinical Network Publishes New Results in Muscle and Nerve
CMAP Assessed as a Potential Clinical Trial Endpoint During CARNI-VAL Trial. Please click here to read more about this study and how CMAP is relevant to SMA.
 
August 19, 2010   CARNI-VAL Clinical Trial Results for Spinal Muscular Atrophy Published from Project Cure SMA Network.
Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy. The paper was published today in the online Journal PLoS ONE. This clinical trial was fully funded by Families of SMA.
 
August 11, 2010   Rosalind Franklin University Researcher Dr. Michelle Hastings Receives NIH Grant to Pursue Work on the Paratek Compounds for Spinal Muscular Atrophy
Dr. Hastings received key pilot funding from Families of SMA to discover that tetracycline-like molecules developed at Paratek can correct SMN2 splicing and enhance production of SMN protein, as well as to begin development of the compounds for potential clinical use in SMA.
 
July 29, 2010   Keirstead Lab Publishes Paper on Functionality of Motor Neuron Progenitor Cells with Funding from Families of Spinal Muscular Atrophy
Please click here to read more about the functionality of this cell population in an animal model of spinal cord injury.
 
July 15, 2010   New Therapeutic Avenues for Spinal Muscular Atrophy in Development by ISIS Pharmaceuticals, Inc. – First SMA Mouse Model Studies Published
Click here to learn more about this drug candidate and publication of recent testing results in a SMA mouse model by researchers at ISIS and Cold Spring Harbor Laboratory.
 
July 7, 2010   Families of Spinal Muscular Atrophy is Proud to Announce the Launch of the Erin Trainor Memorial Conference Scholarship Fund.
Click here to find out how to support a newly diagnosed SMA family.
 
June 29, 2010   Families of Spinal Muscular Atrophy Grants First Audrey Lewis Young Investigator Award to Dr. George Mentis of Columbia University
Click here to see the announcement.
 
June 29, 2010   Quinazoline Program Makes Progress Toward Drug Treatment for Spinal Muscular Atrophy – Families of SMA 2010 Program Update.
Click here to see the article.
 
June 24, 2010   Families of Spinal Muscular Atrophy Collaboration with Nationwide Children’s for Gene Therapy Development.
Click here for program update 2010.
 
June 22, 2010   Families of Spinal Muscular Atrophy and California Stem Cell, Inc. MotorGraft TM Project 2010 Update.
Click here to see the article.
 
June 18, 2010   Progress Made Against Genetic Killer of Infants and Toddlers by University of Utah Researchers
Project Cure SMA Clinical Trials Network is making headway with support of Families of Spinal Muscular Atrophy.
 
June 17, 2010   Families of Spinal Muscular Atrophy Launches New Support Program to Connect Members of the SMA Community.
New SMA Community Connections.
 
June 11, 2010   Families of SMA Fundraising Events Raise Over $605,000 for Spinal Muscular Atrophy Research and Support in Just Two Months!
Click here to see the events.
 
April 27, 2010   Families of Spinal Muscular Atrophy Awards $250,000 in New Contracts to Support Ongoing Clinical Trials for SMA.
Click here for more details.
 
March 24, 2010   Families of Spinal Muscular Atrophy Announces Members of the New Translational Advisory Council (TAC) to Select New Drug Discovery Projects for SMA.
Please click here to read about the TAC members.
 
March 22, 2010   Families of Spinal Muscular Atrophy is Excited to Announce the Creation of the Newly Diagnosed Type II and Type III Care Packages Program.
Families of SMA began sending these new care packages to newly diagnosed families within the United States beginning January 1st, 2010. These care packages are made up of items that have been suggested by SMA families.
 
March 9, 2010   Isis Pharmaceuticals Licenses Intellectual Property Created at the University of Massachusetts and Funded by Families of Spinal Muscular Atrophy.
To Develop Drugs to Treat Spinal Muscular Atrophy.
 
March 8, 2010   Families of Spinal Muscular Atrophy Launches Fundraising Stars Blog.
To recognize the amazing fundraising efforts made by our members nationwide which help advance research for a treatment and cure for SMA. Click here to read the full article.
 
February 28, 2010   Families of Spinal Muscular Atrophy Awards Grant For Gene Therapy Development for Spinal Muscular Atrophy
Click here for details of the grant to Dr. Brian Kaspar at Nationwide Children’s Hospital of The Ohio State University.
 
January 4, 2010   Project Co-Funded by Families of SMA and SMA Europe Shows Embryonic Stem Cell-derived Neural Cells Improve Spinal Muscular Atrophy in Mice
Click here to read more about this paper published in the journal Brain by Dr. Comi's group at the University of Milan.
 

 

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