Onasemnogene abeparvovec-xioi (Zolgensma®)

Covers a single intravenous lifetime dose of onasemnogene abeparvovec (Zolgensma) at 1.1×10^14 vg/kg for pediatric patients <2 years with genetically confirmed bi‑allelic SMN1 mutations; excludes patients ≥2 years, prior Zolgensma recipients, those with anti‑AAV9 titers >1:50, and patients who cannot be shown to have ≤4 SMN2 copies (effectively excluding ≥5 SMN2) or who will continue Spinraza/Evrysdi. Key requirements: documentation of SMN1 bi‑allelic mutation and SMN2 copy number (with assay verification if 4 copies), baseline anti‑AAV9 ≤1:50, baseline labs (LFTs, creatinine, CBC including platelets, troponin‑I), weight for correct vg/kg dosing, specialist prescribing/consultation, and a corticosteroid regimen equivalent to prednisolone 1 mg/kg/day starting 1 day before infusion and continued 30 days.

"Zolgensma is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutati..."