Spinal Muscular Atrophy - Zolgensma

Covers a single one‑time IV dose of onasemnogene abeparvovec‑xioi (Zolgensma) for pediatric patients <2 years with genetically confirmed bi‑allelic SMN1 mutations who meet SMN2 copy‑number criteria (generally ≤3 copies or select validated 4‑copy results); not recommended for patients ≥2 years, those with prior Zolgensma, complete limb paralysis, permanent ventilator dependence, or baseline anti‑AAV9 antibody titer >1:50. Approval requires specialist prescribing, documentation of genetic tests and SMN2 copy number, weight within 14 days, baseline labs (LFTs, platelets, CBC, creatinine, troponin‑I), initiation of systemic corticosteroids (prednisolone 1 mg/kg/day starting 1 day before infusion for 30 days), and specified pre‑ and post‑infusion safety monitoring and attestations (including discontinuation of other SMA disease‑modifying therapies).

"Treatment of spinal muscular atrophy (SMA) — Approve one-time per lifetime dose for pediatric patients less than 2 years of age with genetically confirmed bi-allelic mutations in the survival motor..."