Spinal Muscular Atrophy (SMA) is a hereditary disorder that destroys the nerves controlling voluntary muscle movement. It is the number one genetic killer of children under the age of two. SMA is a relatively common “rare disorder.” It is estimated that SMA occurs in about 1 in every 6,000 births. Approximately 1 in 40 individuals (7.5 million Americans) carry the gene that causes SMA, making it the second most common autosomal recessive genetic disorder. This incidence rate shows neither racial nor gender bias.
Presently, there is no known treatment for SMA, though there have been several exciting research breakthroughs over the past decade. Among more than 600 neurological disorders, SMA has been singled out by the National Institutes of Health as the disease closest to treatment based on scientists’ advanced genetic understanding of the disease.
In order to support the investigators, clinicians, and families who are working to find a treatment or cure for SMA, the SMA community, including Families of SMA, the SMA Foundation, Fight SMA, and the Muscular Dystrophy Association, has united behind the “SMA Treatment Acceleration Act.” This legislation authorizes federal funding in order to:
- Upgrade and unify existing SMA clinical trials sites and establish a national clinical trials network for SMA.
- Establish a Data Coordinating Center to provide expert assistance and advice to SMA clinical trials sites.
- Expand and intensify federally supported research programs with respect to pre-clinical translational research related to SMA.
- Establish a research collaborative at the National Institutes of Health to ensure cooperation across multiple Institutes regarding research related to SMA.
- Enhance and provide ongoing support to the existing SMA patient registry in order to provide for expanded research on the epidemiology of SMA.
- Establish an SMA Coordinating Committee, consisting of representatives from relevant government agencies and the public, to coordinate government activities relating to SMA, serve as the principal advisor to agency heads, and conduct a study to identify barriers to the development of drugs for treating SMA and report findings and legislative recommendations to Congress.
- Require the Secretary of Health and Human Services to collaborate with the FDA and the Coordinating Committee to make recommendations for improving and expanding existing industry incentives to promote SMA drug development.
- Establish and implement a program for providing information and education on SMA to health professionals and the general public related to advances in the diagnosis and treatment of SMA and the provision of care to SMA patients.
The SMA Treatment Acceleration Act provides federal support to complement the substantial private funding that national non-profit organizations are investing to find a treatment for SMA. Passage of this landmark legislation will enable investigators to mount national clinical trials to demonstrate that identified therapeutics are safe and effective for SMA patients.
If you have any questions about the SMA Treatment Acceleration Act, or if you would like information about how you can become a cosponsor, please contact Spencer Perlman of Families of SMA (spencer@fsma.org / 202-333-5750), Laura Breiteneicher of the SMA Foundation (laurab@wswdc.com / 202-589-0800), Caroline Gibson of Fight SMA (carolinegibson@fightsma.com / 804-515-0080) or Annie Kennedy of the Muscular Dystrophy Association (akennedy@mdausa.org /202-828-8560).
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