Neurology – Skysona®

Covers a single, one-time intravenous Skysona infusion for male patients aged 4 to <18 with early, active cerebral adrenoleukodystrophy (NFS ≤1, gadolinium-enhancing MRI, Loes 0.5–9) who have a confirmed ABCD1 mutation, elevated VLCFA, no HLA‑matched family donor, adequate hepatic/renal/hematologic function, negative infectious disease screening, planned G‑CSF mobilization/apheresis with busulfan myeloablative conditioning and cyclophosphamide or fludarabine lymphodepletion, VOD prophylaxis, contraception, specialist prescribing, and a minimum dose of 5.0×10^6 CD34+ cells/kg — excluded are patients with full ABCD1 gene deletion, prior allogeneic HSCT or prior gene therapy, advanced disease (Loes >9 or NFS >1), active infection or disqualifying labs, non‑male patients, or lack of required documentation/claims verification.

"Documentation of absence of active bacterial, viral, fungal, or parasitic infection prior to therapy."