Described as the ‘world's most expensive drug’, Zolgensma is now available on the NHS to treat children with spinal muscular atrophy, the leading genetic cause of death for children
Spinal muscular atrophy (SMA) is a genetic condition that causes weakening of muscles and problems with movement (NHS, 2020). Until 2 years ago, there were no treatments available for children diagnosed with this disease, but a landmark deal between the NHS and Novartis Gene Therapies has made this cutting edge gene therapy available on the NHS (NHS England, 2021).
Zolgensma, which has a list price of £1.795 million per dose, is a gene therapy that aims to target the cause of SMA. SMA is caused by a defect in a gene called SMN1 that produces a vital protein for nerves that control muscle movements (European Medicines Agency, 2021). The treatment uses a virus to deliver a corrected version of the SMN1 gene into the child's body (European Medicines Agency, 2021).
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