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Spinal Muscular Atrophy (SMA)

The most common inherited cause of early childhood death.  SMA destroys nerve cells that affect voluntary movement; therefore symptoms include difficulty breathing, swallowing, controlling head and neck, crawling, and walking (does not affect intelligence).  The most common form affects infants, and can cause death by ages 2-4, although a milder form may allow survival into adulthood.  Unfortunately, there is no treatment or cure.  Inherited in an autosomal recessive pattern, a child has a 25% chance of developing CF if both parents are carriers.  Carried in 1:41 of all people in the U.S.