NHS England strikes deal on gene therapy onasemnogene abeparvoccex (Zolgensma) for treatment of Spinal Muscular Atrophy (SMA)

Zolgensma, with reported list price of £1.79m per dose, will be available to patients at a price that is fair to taxpayers after a deal struck by NHSE. This will allow up to 80 patients a year to benefit from single IV infusion therapy containing a replica of missing gene SMN1.

SPS commentary:

This treatment has been accepted by NHSScotland for use in patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.

Source:

NHS England

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