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Rep. Patrick J. Kennedy

Madam Speaker, today, along with my colleague Representative Eric Cantor, I am introducing the SMA Treatment Acceleration Act, in an effort to help find a treatment or cure for Spinal Muscular Atrophy (SMA), the number one genetic killer of children under the age of two.

SMA is an inherited disease that destroys the nerves controlling muscle movement, which affects crawling, walking, head and neck control, swallowing, and even breathing. The gene mutation that causes SMA is carried by one in every 40 people, or approximately 7.5 million Americans. Each child born of two carriers of the mutant gene has a one in four chance of developing 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. This priority status is 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. This research is providing groundbreaking data for SMA and other neurodegenerative disorders, including the muscular dystrophies, Freidrich's Ataxia, Fragile X syndrome, and Huntington's disease.

This legislation will upgrade and unify SMA clinical trial sites and establish a national clinical trials network for SMA. It will also establish a Data Coordinating Center, expand and intensify federally supported research programs, and promote collaborative research at NIH. Additionally, the bill will enhance and provide support for the SMA patient registry, establish an Interagency SMA Research Coordinating Committee, and establish and implement a program for providing information and education on SMA to health professionals and the general public. These provisions will advance our research and understanding of SMA and lead towards effective treatments. I am proud to be reintroducing this legislation, and I urge your full consideration of this important legislation.