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NHS secures world’s most expensive drug to treat babies with rare muscle disease – The Irish News

Patients will be able to access Zolgensma, a one-time gene therapy, said NHS England.

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Up to 80 babies a year born with a rare muscle-wasting disease could receive life-changing treatment after the most expensive drug in the world was licensed for use on the NHS in England.
Infants born with Spinal Muscular Atrophy (SMA), an often fatal condition which affects the spinal cord nerves causing paralysis, will be able to access Zolgensma, a one-time gene therapy, said NHS England.
The drug, which replicates a missing gene and restores nerve and muscle function, has a reported list price…

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