SMA THERAPIES I

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Nusinersen has demonstrated a favorable benefit:risk profile and shown significant and clinically meaningful efficacy on motor function across a broad spectrum of SMA populations, and event-free survival (EFS; time to death or permanent ventilation) in infantile-onset SMA. The current analyses report interim results from the SHINE study (NCT02594124) for individuals with infantile-onset SMA who transitioned from ENDEAR. SHINE is an open-label extension study for infants/children who participated in the nusinersen clinical trial program.

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