1.8m-per-dose gene therapy approved for NHS use in Scotland

摘要

A one-off treatment for babies with a rare genetic disorder is the first gene therapy to be accepted by the Scottish Medicines Consortium (SMC) for use by NHS Scotland, and could become the most expensive treatment to ever be approved by the National Institute for Health and Care Excellence (NICE) in England and Wales. Onasemnogene abeparvovec (Zolgensma; Novartis Gene Therapies), which costs ￡1.79 million per dose, is a potentially curative therapy for babies with spinal muscular atrophy (SMA); a rare, progressive neuromuscular condition caused by a genetic mutation in the SMN1 gene on chromosome 5q.