The FDA approved Novartis’ intrathecally delivered SMA gene therapy Itvisma (onasemnogene abeparvovec), offering a new delivery route intended to broaden access beyond the intravenous label of Zolgensma. The agency highlighted the benefit of a fixed intrathecal dose for older or heavier patients. Ten days later, Sarepta Therapeutics faced additional FDA restrictions on its DMD gene therapy Elevidys, underscoring the regulatory complexity of neuromuscular gene medicines. The twin developments mark both expansion and tightening of oversight in the gene‑therapy field for neuromuscular indications.