[Congressional Record (Bound Edition), Volume 155 (2009), Part 8]
[Extensions of Remarks]
[Pages 10963-10964]
[From the U.S. Government Publishing Office, www.gpo.gov]




                     SMA TREATMENT ACCELERATION ACT

                                 ______
                                 

                        HON. PATRICK J. KENNEDY

                            of rhode island

                    in the house of representatives

                        Tuesday, April 28, 2009

  Mr. 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

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I urge your full consideration of this important legislation.

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