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Facts about Spinal Muscular Atrophy
  • Spinal Muscular Atrophy (SMA) is the leading genetic killer of infants and toddlers. Each year 1,000 babies are born with SMA in the U.S. and yet so many of us have not even heard of this disease. These infants simply do not get a chance to grow up to be adults, do not get a chance to become famous, and do not make the headlines when they die for the world to hear about this deadly disease.

  • The sad news is . . . Whether we have heard of SMA or not, this ‘little known disease’ is afflicting an estimated 1 in every 6,000 live births. Worldwide, 13,000 lives are lost every year. Over 25,000 Americans have SMA.

  • The good news is . . . The international scientific community agrees that of all neuromuscular diseases, SMA is the closest to developing a treatment or a cure. Because a cure is in sight, the National Institutes of Health (NIH) recently designated SMA as a model for their research program aimed to turn basic science into actual drugs and treatments. It is the educated expectation of leading scientists and clinicians worldwide that with the appropriate funding, an effective therapy for SMA can be achieved in five years or less. Small private investments in SMA research have resulted in tremendous breakthroughs.

  • SMA does not affect sensation and intellectual activity in patients. It commonly is observed that patients with SMA are unusually bright and sociable.


    • Type 1, or Werdnig-Hoffmann Disease

    • Type II SMA affects infants between seven and 18 months old.

    • Type III, also known as Kugelberg-Welander Disease, 

    • Type IV is the adult form of the disease in which symptoms tend to begin after age 35. 

For more information please visit: CURE SMA


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