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.
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 email@example.com. Click the link for more information!
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!
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.
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!
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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).
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.
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.
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.
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.
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.
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.
“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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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).
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.
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.
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.
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.
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.
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.
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.
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.