NHS England agrees deal for gene therapy for spinal muscular atrophyBMJ 2021; 372 doi: https://doi.org/10.1136/bmj.n653 (Published 08 March 2021) Cite this as: BMJ 2021;372:n653
- Elisabeth Mahase
- 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 …