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NHS drugs go-ahead offers lifeline to children with rare muscle-wasting disease

Medicines watchdog approves two treatments for patients with spinal muscular atrophy

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The National Institute for Health and Care Excellence (Nice) has published final draft guidance recommending that any patient who would benefit can have either drug.

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May 13, 2026 Children National Institute for Health and Care Excellence NHS

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