FDA has granted approval to Scholar Rock’s Isembyld (apitegromab), marking the first muscle-targeted treatment for spinal muscular atrophy and arriving after prior manufacturing setbacks. The drug is designed to improve motor function in people with SMA who are already on standard therapies. In parallel, Novartis delivered an update on del-desiran in the Phase III HARBOR trial for myotonic dystrophy type 1 (DM1). The study missed its primary endpoint on video hand opening time (vHOT), though the company reported evidence of clinical activity in secondary endpoints as it evaluates the full dataset with health authorities.