Novartis received FDA approval to expand delivery of its SMA gene therapy (Itvisma) via an intrathecal route, widening patient eligibility and affirming gene-replacement approaches for neuromuscular disorders. At the same time, Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys encountered new FDA restrictions, marking a turbulent week for neuromuscular approvals and access. The contrasting outcomes highlight regulatory nuance around vector delivery, long-term safety data, and benefit-risk calculations. Sponsors of gene therapies should expect close scrutiny of administration routes and post-approval commitments. Clinicians and patient groups will assess how the new SMA approval alters treatment algorithms and access for older or heavier patients previously ineligible for intravenous dosing.