| December 29, 2010 |
Families of Spinal Muscular Atrophy Evaluating Six New Drug Discovery Programs for New Funding in 2011.
An update on the new Translational Drug Discovery Program at FSMA.
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| December 22, 2010 |
Families of Spinal Muscular Atrophy is Requesting New Basic Research Grant Applications for 2011 Funding.
Continued investment in basic research, leading to a greater understanding of the exact nature, causes, and consequences of SMA, is key to ensuring the most effective SMA treatments can be identified and developed as quickly as possible.
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| December 16, 2010 |
Families of Spinal Muscular Atrophy Research Funding Leads to the Publication of 17 Journal Articles in 2010.
Click here to see the details.
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| December 15, 2010 |
Muscular Dystrophy Association Invests $1.4 Million to Help Advance the Families of SMA Quinazoline Drug Program for SMA at Repligen.
Click here to see the details.
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| November 18, 2010 |
Multiple Presentations on Spinal Muscular Atrophy at The 2010 Society for Neuroscience Meeting.
The 40th Annual Society of Neuroscience Meeting was held November 13th to 17th in San Diego, California.
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| November 9, 2010 |
Families of Spinal Muscular Atrophy Announces the VALIANT Clinical Trial of Type III SMA Patients Conducted at The Ohio State University Is Complete.
This study was a randomized placebo-controlled clinical trial designed to test the efficacy and safety of the combined treatment of valproic acid and carnitine in ambulatory adults living with Type III SMA.
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| October 22, 2010 |
The California Institute for Regenerative Medicine Awards Ipierian $5.6M to Advance a Spinal Muscular Atrophy Drug Program
The California Institute for Regenerative Medicine made 19 awards, totaling $67 million yesterday in the second round of a program aimed at pushing stem cell projects out of the lab and into the clinic. This includes a project at iPierian designed to identify small molecules that increase the expression of SMN protein in SMA patients.
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| October 20, 2010 |
Dr. Hans Keirstead Publishes Expert Opinion Article in Expert Opinion in Biological Therapy
In this article, Dr. Keirstead discusses possible ways that Cellular Therapy transplanted into the spinal cord could potentially help motor neurons and synapses by providing neurotrophic support. The benefits of neurotrophic support are contingent on the ability of the transplanted cells to release neurotrophins (neuronal growth factors) that impact degenerating motor neurons in the spinal cord or development/stability of the neuromuscular junction (NMJ).
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| September 21, 2010 |
Live webcast available for the upcoming FDA and NIH co-sponsored meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases.
Please click here to learn more about this meeting and how you can view it live over the internet.
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| August 26, 2010 |
Recent Slate Article Addresses the Challenges of Therapy Development, Including Those Relevant to Spinal Muscular Atrophy
This article discusses balancing hope and realty when developing potential drugs like stem cell and gene therapies. Please click here to read the Slate article.
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| August 11, 2010 |
Three Papers Published in Human Molecular Genetics Describe Heart Defects in a Severe Model of Spinal Muscular Atrophy
Studies by the DiDonato Lab at Northwestern University enabled with FSMA funding and by the Kaspar Lab in Ohio, and by the Lorson lab at the University of Missouri demonstrate arrhythmia and cardiac defects are a common feature in a severe mouse model of Spinal Muscular Atrophy
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| August 2, 2010 |
Dr. Sproule and Colleagues at Columbia University Publish a Paper Showing Fat Mass is Increased in High-Functioning, Non-Ambulatory SMA Patients.
Please click here to read the paper abstract.
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| July 27, 2010 |
Highlights From the 14th Annual SMA Research Group Meeting.
Please click here to learn more about the 2010 SMA Research Group Meeting held in Santa Clara, CA.
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| July 26, 2010 |
Cost of discovery: A glimpse at why research in the life sciences is so expensive
Please click here to read a short news paper article on cost of biology research.
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| July 7, 2010 |
Two Articles on Spinal Muscular Atrophy Newborn Screening in the Latest Issue of the American Journal of Medical Genetics
Dr. Prior at OSU publishes a research article on the technical feasibility of both carrier and newborn screening for SMA. Dr. Swoboda at the University of Utah publishes an editorial on the critical importance of newborn screening in SMA.
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| May 28, 2010 |
Families of SMA Receives Grant from the National Institute of Neurological Disorders and Stroke
To support the 14th Annual SMA Research Conference. We thank them for their fantastic support that has helped bring together the SMA research community along with our families.
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| May 27, 2010 |
Researchers will Participate in a Special Session on Drug Discovery at the 14th Annual SMA Research Group Meeting
FSMA has organized a session at the SMA Research Group Meeting on the required steps to progress a drug candidate to human clinical trials. Click here to read more.
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| April 27, 2010 |
Families of Spinal Muscular Atrophy Funded Quinazoline Program Featured in American Chemical Society Symposium on Rare and Neglected Diseases
Click here for details.
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| April 14, 2010 |
Families of Spinal Muscular Atrophy Builds a Drug Pipeline for SMA as Described in the Latest Issue of “Compass”
Click here to download a copy.
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| April 7, 2010 |
Multiple Presentations on Spinal Muscular Atrophy at The 62nd American Academy of Neurology Meeting.
Including Five by the Project Cure SMA Clinical Trial Network Funded by Families of SMA.
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| April 7, 2010 |
Over 100 Presentation Requests Submitted to the 14th Spinal Muscular Atrophy Research Group Meeting Hosted by FSMA
The conference, which hosts about 250 researchers, will be held in Santa Clara, California from June 24th to 26th, 2010.
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| February 22, 2010 |
Families of Spinal Muscular Atrophy Announces New Translational Advisory Council (TAC) to Select and Govern New Drug Discovery Projects.
Click here for details.
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| January 25, 2010 |
Families of SMA Funded Study Shows the HDAC Inhibitor SAHA Ameliorates the Spinal Muscular Atrophy Phenotype in Two Mouse Models of SMA.
Lead author Dr. Riessland in the laboratory of Dr. Brunhilde Wirth publishes paper in Human Molecular Genetics.
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| January 18, 2010 |
Families of Spinal Muscular Atrophy Funding Leads to Publication of New Clinical Trial Outcome Measure for Type I Infants
"The Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND)" Published in the Journal Neuromuscular Disorders
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| August 20, 2010 |
Spinal Muscular Atrophy Satellite Symposium at Society of Neuroscience
SMA groups to hold symposium at the Society for Neuroscience Meeting entitled "Nucleic Acids to the Rescue: Gene and Antisense Oligonucleotide Therapies for SMA".
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