Spinal muscular atrophy
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Spinal muscular atrophy (SMA) is a serious disease that causes nerves and muscles to stop working. Babies affected by SMA often get weak and might die after their lungs fail, and if disease develops in older children or adults they will gradually lose the ability to move their bodies.
Spinal muscular atrophy is inherited from parents who might or might not have the disease themselves but both of whom have a fault (mutation) in their genes that predisposes to the disease. Around one in 25 to one in 50 persons, depending on the country, carries a faulty gene of SMA.
Spinal muscular atrophy is the most common genetic disease causing death of babies. Only one medicine helps in SMA, it is called Spinraza.