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 ...
At a mean age of 9 years old, ELEVIDYS-treated patients achieved mean North Star Ambulatory Assessment (NSAA) scores above baseline three years after treatment (n=52) ELEVIDYS gene therapy ...
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