We are very pleased to announce that legislation to enhance federal support for Spinal Muscular Atrophy (SMA) research, H.R. 3334, “The SMA Treatment Acceleration Act”, has been introduced in the U.S. House of Representatives by Congressman Patrick Kennedy (D-RI) and Congressman Eric Cantor (R-VA). U.S. Senator Debbie Stabenow (D-MI) and Senator Johnny Isakson (R-GA) plan to introduce companion legislation in the Senate when Congress returns from its August district work period. On behalf of our organizations and the families affected by this deadly disease, we want to express our gratitude and thanks to Congressmen Kennedy and Cantor and Senators Stabenow and Isakson for their leadership.
SMA is the number one genetic killer of children under the age of two. It is an inherited disease that destroys the nerves controlling muscle movement, which affects crawling, walking, head and neck control, swallowing, and even breathing. Approximately one in 40 people, or approximately 7.5 million Americans, carry the gene mutation that causes SMA. Each child of two carriers of the mutant gene has a one in four chance of being afflicted by SMA.
Among more than 600 neurological disorders, SMA has been singled out by the National Institutes of Health (NIH) as the disease closest to treatment based on scientists’ advanced genetic understanding of the disease and a strong collaboration between families, federal agencies, and patient advocacy groups. Researchers have identified the gene responsible for SMA, as well as a disease modifying “back-up” gene that has opened the door to promising new treatment pathways. Research involving the modulation of genes stands to benefit not only SMA, but other genetic disorders, including Duchenne Muscular Dystrophy, Parkinson’s, and Alzheimer’s. In recent Congressional testimony by the NIH, it was noted that “research on SMA illustrates the path from gene to understanding to treatment.”
In order to build on the progress being made by investigators and bring treatments to children affected by SMA, a broad coalition of organizations, including FightSMA, Families of SMA, and the SMA Foundation, has united behind the SMA Treatment Acceleration Act, legislation aimed primarily at supporting a national clinical trials network for SMA.
Specifically, “The SMA Treatment Acceleration Act” provides for the following:
- Federal support for a national clinical trials network for SMA;
- Federal support to enhance the existing SMA patient registry and for expanded research on the epidemiology of SMA;
- Establishes an SMA Coordinating Committee to include federal agencies, SMA researchers, and SMA families, which shall study barriers to development of SMA treatments;
- Establishes a trans-Institute research collaboration at NIH under the Director to ensure that all relevant Institutes are contributing and collaborating on SMA research;
- Requires the Secretary of the U.S. Department of Health and Human Services (HHS) to study and report to Congress on the use of incentives to promote SMA drug development among private industry;
- Provides for the Secretary of HHS to establish a program to provide information and education on SMA to health professionals and the general public
Our organizations will be issuing a “Nationwide Call to Action” for all SMA families, researchers, and friends, to help engage every Member of Congress in support of the this bill and the great efforts of Congressmen Kennedy and Cantor and Senators Stabenow and Isakson.
Cynthia Joyce
SMA Foundation
Kenneth Hobby
Families of SMA
Martha Slay
FightSMA
NOTE: For more information on the “The SMA Treatment Acceleration Act” please contact any one of our Government Affairs staff:
Laura Breiteneicher
SMA Foundation
laurab@wswdc.com
Spencer Perlman
Families of SMA
spencer@fsma.org
Caroline Gibson
Fight SMA
carolinegibson@fightsma.com
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This episode is a bit of a departure for the Fighting Back Podcast. We usually interview people and families fighting against serious diseases. We’ve spoken to a woman trying to raise the profile of SMA through a one-woman stage show, a mother starting a foundation to raise money for myotonic muscular dystrophy, a mother who launched what became a national organization to fight for orphan disease, and many more. This episode is a bit different, however. This time we’re talking to a man who is fighting diseases directly.
