About legislation H.R. 2149, S. 1158 (111th Congress):
Investigators, clinicians, and families are working to find a treatment or cure for spinal muscular atrophy (SMA). The SMA Treatment Acceleration Act supports these efforts with federal funding. The legislation authorizes federal funding in order to:
- Upgrade and unify 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 preclinical translational research related to SMA.
- Enhance the SMA patient registry for expanded research on the epidemiology of SMA.
- Establish an Interagency SMA Research Coordinating Committee, consisting of representatives from relevant government agencies and the public, to coordinate government activities relating to SMA, develop a comprehensive strategy for improving and expanding SMA research, make recommendations to strengthen collaborative research across multiple institutes at National Institutes of Health (NIH), and identify barriers to the development of drugs for SMA treatments.
- Promote collaborative research at the NIH to ensure cooperation across multiple institutes regarding SMA-related research.
- Establish and implement a program for providing information and education on SMA to health professionals and the general public related to advances in the screening, diagnosis and treatment of SMA, and the provision of care to SMA patients.
Spinal muscular atrophy (SMA) is a hereditary disorder that destroys the nerves controlling voluntary muscle movement. It is the No. 1 genetic killer of children under age 2. 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) carries 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, but there have been several exciting research breakthroughs over the past decade. Among more than 600 neurological disorders, SMA has been identified by the National Institutes of Health as being one of the diseases closest to treatment based on scientists' advanced genetic understanding of the disease.
The SMA Treatment Acceleration Act provides federal support to complement the substantial private funding that national nonprofit 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 people with SMA.
In the previous Congress, similar legislation was introduced and received terrific support. The House bill was sponsored by Rep. Patrick Kennedy, D-R.I., and had 70 co-sponsors, including 49 Democrats and 21 Republicans. Sen. Debbie Stabenow, D-Mich., sponsored the Senate bill which had 19 co-sponsors (15 Democrats, two Independents and two Republicans).
On the record:
- House Dear Colleague Letter from Rep. Kennedy and Rep. Cantor
- Senate Dear Colleague Letter from Sen. Stabenow and Sen. Isakson
- Scientist and Clinician Letter of Support for the SMA Treatment Acceleration Act to Sen. Stabenow and Sen. Isakson
Support Letters from the SMA Government Relations Team:
- SMA Grassroots Update - 10/26/2010
- SMA Grassroots Update - 4/20/2010
- SMA Grassroots Update - 11/2/2009
- SMA Grassroots Update - 7/24/2009
- SMA Grassroots Update - 6/26/2009
- SMA Grassroots Update - 5/26/2009
- SMA Grassroots Update - 4/29/2009