NewsDialy

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

9/13/2026

Federal regulators approved a drug on Friday for spinal muscular atrophy, a rare neurological condition where the nerve cells that signal muscles to move gradually deteriorate.

The drug, Isembyld, is the first approved therapy to directly address the muscle loss the disease causes.

Scholar Rock, the company behind it, received Food and Drug Administration clearance for use in adults and children aged 2 and older who are already receiving SMA treatments that work on a gene called SMN2.

The approval matters for patients because existing SMA drugs cover only part of what the disease does.