In FIREFISH Part 1, efficacy for infantile-onset SMA was established based on the ability to sit without support and survival without permanent ventilation

FIREFISH is an open-label, 2-part study to investigate the efficacy, safety, and tolerability of Evrysdi in infants with Type 1 SMA. Learn more about the FIREFISH trial design.

Sitting milestone

Survival data

Additional data

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