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What is SMA?

Kennedy at Physical TherapySpinal Muscular Atrophy (SMA) is the number one genetic killer of children under the age of two, but have you ever heard of it? SMA is an inherited and often fatal disease that destroys the nerves controlling voluntary muscle movement, which affects crawling, walking, head and neck control, and even swallowing. It is in the family Muscular Dystrophies which involves muscles that become weak and lose muscle tissue over time.

Facts About SMA

  • Two new-born children are affected by SMA each day in the United States, approximately 1 in 6,000 live births.
  • One in every 40 people carries the gene that causes SMA, which means there are more than seven million carriers in the United States alone.
  • If both parents are carriers of the gene that causes SMA, there is a 25% chance the baby will inherit the recessive gene from each parent and develop this disease.

Currently there is no treatment or cure. However, given the remarkable advances of the past 15 years, scientists now consider SMA to have the greatest probability of realizing a treatment or cure than any other major genetic disease. However, development of these treatments within the next few years will likely require $20-30 million or more of sustained annual research funding.

SMA Research
The Families of SMA website states there are currently three research areas associated with SMA:

  • Basic Science Research
  • Drug Discovery/Transitional Research
  • Clinical Trials

"Basic research in SMA biology tells us what causes SMA. Understanding what causes SMA reveals new and more effective ways of making SMA drugs. Pre-clinical drug discovery programs then convert basic research ideas into novel kinds of SMA drug candidates. Establishing clinical trial infrastructure provides the means to test the drug candidates in humans. Therefore, continued investment in all three research areas is essential to finding an effective SMA treatment and ultimate cure." (Families of SMA)

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