The FDA approved Novartis’ intrathecal version of its SMA gene therapy, expanding access pathways for spinal muscular atrophy patients by enabling direct spinal delivery. The approval follows data supporting efficacy across a broader age and symptom range and offers an alternative to intravenous dosing that can be limited by systemic exposure concerns. In the same window of neuromuscular industry news, Sarepta encountered a regulatory setback for its Duchenne muscular dystrophy gene therapy program, prompting reevaluation of its clinical and regulatory strategy. The juxtaposition of a new approval and a high‑profile setback highlights the volatile, high‑stakes nature of gene‑therapy development in neuromuscular diseases. Companies and regulators will use these outcomes to refine trial design, delivery routes and benefit‑risk assessments for future gene‑replacement programs.