The global study met its primary endpoint of infants sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). Type 1 SMA babies do not achieve this milestone without therapy. Substantial results were demonstrated across multiple secondary and exploratory endpoints, as infants in the study achieved milestones not seen in natural history. In addition to being able to sit without support, infants were able to maintain upright head control, roll to the side, and stand without support after 12 months of treatment. At 12 months, 95% infants maintained the ability to swallow and 89% were able to feed orally. No new safety signals were identified in part 2 of the study.
'The positive results from part 2 of the FIREFISH study reinforce the clinically meaningful efficacy seen in part 1 of the trial and support its overall safety profile,' said
The single-arm part 2 of FIREFISH assessed the efficacy of risdiplam in 41 infants (eligible age at enrollment between 1 and 7 months) with type 1 SMA treated for 12 months. The study met its primary endpoint with 29% of infants (12/41; p
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