Roche?s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA)
- FIREFISH Part 2 study met its primary endpoint by demonstrating a significant increase in motor milestones in infants aged 1-7 months after 12 months of treatment
- Large, pivotal global study confirms clinically meaningful efficacy seen in the dose-finding Part 1 of the trial
- Safety was consistent with the safety profile observed to date and no new safety signals were identified
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