Apitegromab Adds Muscle-Targeted SMA Treatment

FDA approved Scholar Rock's apitegromab (ISEMBYLD) as an add-on to SMN2-directed therapy for SMA after randomized phase 3 evidence showed a 2.2-point HFMSE advantage and more meaningful motor improvement. Fracture rates, durability, age-group generalizability, access, infusion burden, and real-world functional benefit remain important uncertainties.