Scholar Rock wins FDA approval for muscle-targeted SMA treatment
Scholar Rock ($SRRK) received FDA approval for ISEMBYLD, its apitegromab treatment for spinal muscular atrophy, in patients aged two and older receiving SMN2-targeted therapy. The company plans to begin shipping the medicine within days. The decision moves its muscle-targeted approach from clinical development into commercial use and also brings a rare pediatric disease priority review voucher. The supporting SAPPHIRE study assessed motor function alongside existing treatment. In the specified 1
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