Spinal Muscular Atrophy (SMA)
About Spinal Muscular Atrophy (SMA)
SMA is estimated to occur in 1 in 6,000 to 1 in 10,000 live births3. Its severity correlates with the amount of SMN protein. People with Type 1 SMA, the most severe life-threatening form, produce very little SMN protein and don’t live beyond two years old without respiratory support. People with Type 2 and Type 3 SMA produce greater amounts of SMN protein and have less severe, but still life-altering forms of SMA2.
1. FDA approves first drug for spinal muscular atrophy,
FDA, Dec 2016.
3. Spinal muscular atrophy,
D'Amico, A., et al., Orphanet Journal of Rare Diseases, 2011.