NICE recommends routine NHS access to SMA treatments.
NICE has published final draft guidance recommending nusinersen and risdiplam for certain people with spinal muscular atrophy (SMA) in England.
Key points:
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The medicines were previously available through managed access agreements while additional evidence was collected.
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NICE says the treatments can improve survival, slow disease progression and help patients maintain independence and quality of life.
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Data from the managed access period also reported fewer emergency hospital admissions and benefits for carers.
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The guidance supports access to both therapies, allowing clinicians, patients and families to choose the option best suited to individual needs.
SMA is a rare inherited condition that can cause progressive muscle weakness and, in its most serious forms, early death.
