- Hundreds more children suffering from spinal muscular atrophy (SMA) in England are set to gain access to "life-changing" treatments, nusinersen (Spinraza) and risdiplam (Evrysdi), following their approval for routine use on the NHS.
- The National Institute for Health and Care Excellence (NICE) has endorsed the treatments, concluding that they could offer substantial benefits by improving survival rates, slowing disease progression, and helping individuals maintain their independence.
- These drugs were previously available through a special access programme, with NHS England reporting that they have already helped 73 children with severe SMA (Type 1) survive to age five or older, including Ezra Thorman whose mother stated the treatment "fundamentally changed the course of his life".
- SMA is a genetic condition causing severe muscle weakness that can affect breathing, swallowing, and movement, with babies suffering from the most severe form often not expected to live beyond two years without treatment.
- The approval coincides with a new study set to screen approximately 750,000 newborns in England for SMA, assessing the feasibility of adding it to post-birth baby checks, a measure campaigned for by pop star Jesy Nelson after her twins were diagnosed with the condition.
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