While gene therapies have revolutionized treatment for patients with neuromuscular disorders such as spinal muscular atrophy, they are frequently associated with very high costs. Tofersen, a genetic therapy approved by the Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS) associated with a genetic mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS), is currently under review for use in the UK. With the upcoming National Institute for Health and Care Excellence (NICE) decision, Pamela Shaw, DBE, MBBS, MD, FRCP, FMedSci, FAAN, FANA, FAAAS, University of Sheffield, Sheffield, UK, discusses the appraisal route and cost-effectiveness of tofersen in the UK. This interview took place at the 18th Annual Congress on Controversies in Neurology (CONy 2024) in London, UK.
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