Scholar Rock SMA drug approved by FDA

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September 14, 2026↗ source

BioPharma Dive reports the FDA approved Scholar Rock's Isembyld (apitegromab), the first muscle-targeted treatment for spinal muscular atrophy. The drug blocks myostatin, a protein that limits muscle growth, and is cleared for patients 2 and older alongside existing SMN2-targeted therapies. It is the first SMA therapy aimed directly at muscle loss rather than the underlying genetic mechanism.

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