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NHS England agrees deal for gene therapy for spinal muscular atrophy

BMJ 2021; 372 doi: (Published 08 March 2021) Cite this as: BMJ 2021;372:n653
  1. Elisabeth Mahase
  1. The BMJ

NHS England has agreed a deal to ensure that Zolgensma (onasemnogene abeparvovec), a one-off gene therapy medicine, will be made available for patients with spinal muscular atrophy.

The drug, labelled the most expensive in the world at £1.79m a dose (€2.09m; $2.48m), will be sold to the NHS at a discounted rate, although the details …

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