April 28, 2020 / 5:32 AM / a month ago

Roche says risdiplam effective in spinal muscular atrophy trial

ZURICH, April 28 (Reuters) - 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.

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. (Reporting by Michael Shields, editing by John Miller)

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