Thank you, Dr. Greene, for bringing much needed awareness to rare diseases. Thousands of children are dying every year from diseases that receive little attention and limited funding. Our 16-month-old daughter is one of them. Gwendolyn was born perfectly healthy October 4, 2007. Unfortunately, at 9 weeks old she became very ill and was eventually diagnosed with the relatively common "rare" disease Spinal Muscular Atrophy or SMA. Although few have heard of SMA, SMA is actually the #1 genetic killer of infants and toddlers. In fact, 1 in 40 people unknowingly carry the gene responsible for SMA. It is terminal. It is degenerative. It is cruel. SMA results in the loss of nerves in the spinal cord and the weakness of the muscles connected with those nerves. The prognosis is dire, with a life expectancy of between one and two years. Gwendolyn will never walk, never sit up unassisted, and spends most of her time completely flat where she is most comfortable. Some days we can not even pick her up and snuggle because the movement is too difficult for her. She needs assistance to breathe and even to swallow her own saliva. She may never speak, although we are hopeful. And, while she currently has some arm movement, it seems to weaken every day. However, her mind will always remain unaffected and already she wants so desperately to do all the things that her failing body hinders.

Although, Gwendolyn's disease currently has no treatment and no cure, the National Institutes of Health (NIH) has described SMA as the disease "closest to treatment" and researchers claim they are just a few years away from finding a cure. And, there is landmark legislation, the SMA Treatment Acceleration Act, currently going before Congress that, if passed, would provide researchers the resources needed to make that last crucial step. This is exciting for families impacted by SMA, but this legislation has far greater ramifications. SMA research has already benefited the research of other diseases, such as ALS/Lou Gehrig's, Parkinson's, Alzheimer's, Duchenne Muscular Dystrophy, Tay Sachs, Fragile X, and many, many others. In fact, it is because researchers know so much about SMA that the national organizations consider it a "model" disease from which so much can be learned and put toward saving countless lives.

Having been initially told that there was nothing we could do but go home and love our baby (an impossible task), it is empowering to be so close to changing this outlook and, perhaps, saving the lives of thousands of children. Thus, we joined the battle being waged by the SMA community nationwide and created an online petition – PetitionToCureSMA.com – as a grassroots effort to drum up broad national support for the SMA Treatment Acceleration Act. And it is making a difference. We currently have over 50,000 signatures and Congress is taking notice. However, because SMA awareness is almost nil, the children impacted by this debiliating disease will not receive adequate attention without the efforts of many.

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