Guardian UK5/14/2026Health4 min readUnited Kingdom
NHS to Provide Lifesaving Drugs for Children with Rare Muscle-Wasting Disease
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NICE recommends NHS funding for nusinersen (Spinraza) and risdiplam (Evrysdi) for spinal muscular atrophy (SMA) patients in England, Wales, and Northern Ireland, offering improved survival and quality of life for hundreds of children with this rare genetic disorder.
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Hundreds of children with a rare muscle-wasting disease will be able to receive two drugs that can improve their survival in a move parents hailed as a “lifeline”. The National Institute for Health and Care Excellence (Nice) has published final draft guidance recommending that any patient who would benefit can have either drug. ... (Full article text preserved with paragraph breaks)
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This article was originally published by Guardian UK.
