'Life-changing' SMA treatments approved for routine NHS use offer hope to thousands

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Two groundbreaking treatments for the muscle-wasting condition, spinal muscular atrophy (MSA), have secured approval for routine NHS use in England, offering hope to hundreds of children affected by this rare genetic disorder.The National Institute for Health and Care Excellence has given the green light for nusinersen and risdiplam to be made widely available, marking a significant milestone for families who have long campaigned for permanent access to these therapies.Both medications were previously accessible only through special schemes while health officials gathered additional evidence on their effectiveness.One mother whose child received early treatment described how it had "fundamentally changed the course of his life".

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TRENDING Stories Videos Your Say NICE's final draft guidance confirms the drugs can enhance survival rates, slow the progression of the disease, and help patients retain their independence.Spinal muscular atrophy is a genetic condition that causes severe...

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