Scholar Rock's muscle-targeting SMA therapy Isemblyd lands FDA approval

The FDA approved Scholar Rock's Isemblyd, the first muscle-targeted therapy for spinal muscular atrophy (SMA), for patients aged two and older already on SMN2 therapy. This approval follows positive Phase III trial results showing improved motor function and allows Scholar Rock to broaden access and support programmes in the US.

This Wire brief sits within Fusion42's coverage of Biotech, and 9 sources have reported it between 12 Sep 2026 and 14 Sep 2026.