7-25-06

Christy Greene

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Dear Senator Boxer:

Spinal Muscular Atrophy (SMA) is a term given to a group of inherited diseases that destroy the nerves controlling voluntary movement such as crawling, walking, head and neck control and even swallowing. It is the primary genetic killer of children under the age of two, and one of the most prevalent genetic disorders overall. This disease discriminates against no one by age, sex or ethnicity and an astounding seven million Americans carry the gene that causes SMA. This debilitating disease causes personal hardship, emotional distress and economic costs on the entire family and community.

There have been great strides in recent SMA Research. Some of these include: The successful mapping and cloning of the SMA gene, identifying the SMN2 protein, developing carrier testing, creating multiple mouse models, identifying a pathway of restoring full length SMN2 and identifying compounds through high throughput screening.

Because of these accomplishments, NINDS is looking at SMA as the model for their translational program. Now more than ever money and support is needed to move basic science into clinical trials. As your constituent I urge you to increase funds for SMA research. 10 million dollars a year for the next five years will put us all closer to the end of this needless suffering and death of thousands.

My daughter Sara has Spinal Muscular Atrophy type 1 which is the most severe form of this devastating disease.  Sara is now three years old and is vent dependent. Sara receives help from the state of California with her medical needs that include;16 hours of LVN care a day, CCS, dme supplies, medi-cal insurance and etc. Some of the doctors we visit regularly are a neurologist, GI doctor, pulmonologist, pediatrician, orthopedic, and various therapists. We would love to avoid spending a lot of our time visiting doctors, therapists, and home health teachers from the school district.  Sara has a beautiful spirit and bright mind and would love to be able to play and interact with other children.  This could happen with scientific breakthroughs in SMA.  The NIH needs money for research and clinical trials.  With research and treatments maybe Sara could have a bit of a more normal life.  Also, if a cure is discovered the state may not have to help pay for her costly medical needs.  We would love for Sara to gain strength to breathe on her own, to speak, and to use her hands to access the world around her.

Please support funding for Spinal Muscular Atrophy!    

Thank you,