[Congressional Record Volume 153, Number 135 (Wednesday, September 12, 2007)]
[Senate]
[Pages S11510-S11511]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

      By Ms. STABENOW (for herself, Mr. Isakson, Mr. Warner, and Mr. 
        Whitehouse):
  S. 2042. A bill to authorize the Secretary of Health and Human 
Services to conduct activities to rapidly advance treatments for spinal 
muscular atrophy, neuromuscular disease, and other pediatric diseases, 
and for other purposes; to the Committee on Health, Education, Labor, 
and Pensions.
  Ms. STABENOW. Mr. President, today I am pleased to introduce the SMA 
Treatment Acceleration Act. I also thank my colleagues, Senators 
Isakson, Warner, and Whitehouse, for joining me in sponsoring this 
important legislation.
  In April, I met with Malorie Fox, a beautiful 4-year-old from Ada, 
Michigan, and several other Michigan families about Spinal Muscular 
Atrophy, SMA, the number one genetic killer of children under 2 years 
of age. SMA is a degenerative disease that weakens the body's muscles 
until they can no longer function, that includes the ability to 
breathe.
  Sadly, Malorie was diagnosed with SMA shortly before her first 
birthday. Her parents were told by her doctors that most children 
diagnosed with SMA never reach this milestone. Thankfully, Malorie 
survived, and with her parents Michelle and James, she continues to 
fight this disease. On her homepage, Malorie wrote: ``My mommy & daddy 
focus on the things I CAN do, not those that I cannot.''
  Malorie and her family are not alone. 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. In 
fact, the National Institutes of Health singled out SMA from more than 
600 neurological disorders as the disease closest to treatment based on 
scientists' advanced genetic understanding of the disease. Private 
foundations and national nonprofit organizations dedicated to finding a 
cure for SMA have also made substantial financial contributions.
  To support the investigators and families who are working to find a 
treatment or cure, the SMA community, including Fight SMA, Families of 
SMA, and the SMA Foundation, has united behind this legislation. This 
bill will provide a roadmap and federal funding to better coordinate 
and facilitate SMA research and treatment. Additionally, the 
legislation will establish a program to provide 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.
  Next Monday is Malorie's birthday, and I couldn't wish for anything 
more for her birthday than a cure for SMA. This legislation will be an 
important step forward in fulfilling that wish. I urge my colleagues to 
join with us in passing it.
  I ask unanimous consent that letter of support be printed in the 
Record.
  There being no objection, the material was ordered to be placed in 
the Record, as follows:

                           Spinal Muscular Atrophy Foundation,

                                               September 12, 2007.
     Hon. Debbie Stabenow,
     U.S. Senate,
     Washington, DC.
       Dear Senator Stabenow: We write to express our strong 
     support for the SMA Treatment Acceleration Act, your 
     bipartisan legislation to help find a treatment or cure for

[[Page S11511]]

     Spinal Muscular Atrophy (SMA), the number one genetic killer 
     of children under the age of two.
       Our organizations support cutting edge SMA research and 
     represent thousands of families across the country that have 
     been affected by SMA, 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.
       These are hopeful times for families affected by Spinal 
     Muscular Atrophy. Researchers have discovered the gene 
     responsible for SMA, opening the door to promising new 
     treatments. SMA was selected by the National Institutes of 
     Health (NIH) as the prototype for an accelerated drug 
     discovery effort, singling out SMA as the disease closest to 
     treatment of more than 600 neurological disorders.
       In order to build upon the substantial investment made by 
     national non-profit organizations and the progress being made 
     by researchers towards bringing treatments to children 
     affected by SMA, our organizations are united behind the SMA 
     Treatment Acceleration Act. This legislation would authorize 
     critical funding in order to upgrade and unify existing SMA 
     clinical trial sites to establish a clinical trials network 
     for SMA; enhance and provide ongoing support to the existing 
     SMA patient registry; establish an SMA coordinating committee 
     consisting of representatives from relevant government 
     agencies and the public; establish an SMA research 
     collaborative at NIH to ensure cooperation across multiple 
     Institutes; and support efforts to identify barriers to drug 
     development and recommend steps to expand existing industry 
     incentives to promote SMA drug development.
       We thank you for your leadership in the effort to conquer 
     this terrible disease, and we look forward to working with 
     you to enact this important legislation.
           Sincerely,
     Cynthia Joyce,
       SMA Foundation.
     Kenneth Hobby,
       Families of SMA.
     Martha Slay,
       FightSMA.
                                 ______