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.
<p>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.</p>