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FDA approves first drug to target muscle loss in spinal muscular atrophy

As of September 2026: Won first FDA approval with Isembyld

Patients with spinal muscular atrophy (SMA) have had drugs that keep motor neurons alive for a decade. None stopped the muscle wasting that defines advanced disease. On September 11, 2026, the FDA approved the first treatment built to do that.

Updated Sep 16