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STAT+: Scholar Rock wins FDA approval for first drug to target SMA muscle loss
A late-stage clinical trial found that, when combined with an SMN2-targeting drug, Isembyld caused young patients’ motor skills to improve after a year. Scholar Rock on Friday received Food and Drug Administration approval for the first-ever therapy that targets the loss of muscle in spinal muscle atrophy, raising hopes that patients with the rare neurological disorder might have a better chance of moving and walking independently. The FDA approv…
