Untreated Spinal Muscular Atrophy leads to severe complications and declines in quality of life, emphasizing the need for early intervention. Natural disease progression when SMA is left untreated ...

-- Unprecedented improvement compared to baseline and natural history in multiple functional measures including Time to Rise from Floor (TTR), 4-Stair Climb (4SC), Performance of Upper Limb (PUL) and ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced ...