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Treatments & Research
Treatments & Research
Although there’s currently no cure for SMA, drug treatments are now available.
These pages tell you about any NHS-funded drug treatments, any in the pipeline, and other related topics. Whatever the treatment, it remains vital that anyone who has SMA also has appropriate care and management based on assessment by and discussion with their clinical team. Though some recommendations are now changing, the internationally agreed Standards of Care for SMA remains a useful reference point.
Interim Results from Risdiplam Jewelfish Trial Published
NHS England agrees to allow access to Sapphire Trial to enhance treatments for SMA
England’s Managed Access Agreement for Nusinersen extended to July 2025
Scholar Rock presents latest TOPAZ Trial data at the international scientific congress on SMA
Food and Drug Administration (FDA) in U.S. approves Evrysdi® (Risdiplam) to include babies under 2 months old with SMA
