Efficacy and safety of risdiplam in patients with type 1 spinal muscular atrophy: a 3-year open-label extension of the two-part, phase 2 FIREFISH trial.

In 30 seconds

This study reports the long-term safety and efficacy of risdiplam in children with type 1 spinal muscular atrophy over a 3-year open-label extension of the phase 2 FIREFISH trial. Among 55 children who continued treatment, 90% were alive at year 5, and 80% were alive without permanent ventilation. The study design was a multicentre, open-label clinical trial.

Key findings

• 90% of children were alive at year 5, with 80% alive without permanent ventilation.
• 29% of children did not require respiratory support at year 5.
• 62% of children could sit without support for at least 5 seconds by year 5.
• No children could walk independently by year 5.

Why it matters

The findings highlight the potential of risdiplam to improve survival and motor function in young children with type 1 spinal muscular atrophy, suggesting a meaningful impact on long-term outcomes in this population.