Health
Babies with rare muscle disease can now get ‘life-changer’ drug on the NHS – BBC Focus Magazine
Patients with spinal muscular atrophy will be able to access Zolgensma, a one-time gene therapy, said NHS England.
Up to 80 babies a year are born with a rare muscle-wasting disease and now they 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…
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