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Interim Results from Risdiplam Jewelfish Trial Published

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Treatments & Research
Image shows test tubes in a science laboratory.

NHS England agrees to allow access to Sapphire Trial to enhance treatments for SMA

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Treatments & Research

England’s Managed Access Agreement for Nusinersen extended to July 2025

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Treatments & Research

Scholar Rock presents latest TOPAZ Trial data at the international scientific congress on SMA

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Treatments & Research

Food and Drug Administration (FDA) in U.S. approves Evrysdi® (Risdiplam) to include babies under 2 months old with SMA

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Treatments & Research