The FDA approved Scholar Rock’s first therapy specifically targeting muscle loss in spinal muscular atrophy, Isembyld, expanding options beyond SMN2-targeted care. The approval covers adults and children age 2 years and older who are receiving SMN2 therapies. In the late-stage program, Isembyld combined with an SMN2-targeting drug improved motor skills after one year compared with placebo plus the SMN2 regimen, with the difference reaching statistical significance. The decision marks a new clinical milestone in myostatin-inhibition approaches for SMA. For developers in neuromuscular disease, the approval underscores momentum for mechanism-diverse combinations built around both motor function and muscle preservation. It also signals that regulators are increasingly willing to support add-on indications where functional endpoints can be demonstrated against a declining trajectory. (Story coverage source: STAT+.)