NHS Approves Two Lifeline Drugs for Children with Spinal Muscular Atrophy
The National Institute for Health and Care Excellence (NICE) has published final draft guidance approving two treatments, nusinersen (Spinraza) and risdiplam (Evrysdi), for patients with spinal muscular atrophy (SMA) in England, Wales, and Northern Ireland. This decision provides a critical lifeline to hundreds of children suffering from this rare, progressive genetic disorder that causes severe muscle weakness and can be fatal in early childhood. Previously available only through limited access schemes since 2019 and 2021 respectively, these drugs will now be routinely available on the NHS to any patient who would benefit. Nusinersen is administered via spinal injection, while risdiplam is taken orally. The approval has been hailed by parents and medical officials as a transformative step, offering hope for improved survival and quality of life. For instance, nine-year-old Ezra Thorman, treated with nusinersen since infancy, defied a life expectancy of under two years and now attends mainstream school. Additionally, the NHS plans to evaluate newborn screening for SMA starting in October, potentially integrating it into routine care to enable earlier intervention.
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