Zolgensma®: gene therapy for spinal muscular atrophy approved for use in the UK

Written by Alice Bough

The National Institute for Health and Care Excellence (NICE; UK) has published draft guidelines on the use of onasemnogene abeparvovec (Zolgensma®) for the treatment of spinal muscular atrophy (SMA) in the NHS in England and Wales. The Scottish Medicines Consortium (Glasgow, Scotland) has also approved the use of the gene therapy, which is made by Novartis Gene Therapies (formerly AveXis). “SMA is a very serious, debilitating and distressing condition that has very significant effects on every aspect of life of those with SMA, and their families and carers,” explained Meindert Boysen (Centre for Health Technology Evaluation at NICE). The drug...

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