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Roche Holding AG’s risdiplam showed significant improvement in survival and motor milestones in a clinical trial for infants with type 1 spinal muscular atrophy (SMA), the Swiss drugmaker said on Tuesday.
Location of neurons affected by spinal muscular atrophy in the spinal cord
The 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, it said. Safety was consistent with the safety profile observed to date and no new safety signals were identified, it added.
