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Risdiplam’s a catch in FIREFISH trial
July/August 2020
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SOUTH PLAINFIELD, N.J.—Part two of PTC Therapeutics Inc.'s FIREFISH study of risdiplam in infants with type 1 spinal muscular atrophy (SMA) yielded strong results in Q2 2020. The study met its primary endpoint of infants sitting without support for five seconds by 12 months, a milestone that infants with SMA generally do not reach without therapy. Ninety-five percent of infants maintained the ability to swallow at 12 months, and 89 percent were capable of feeding orally. Generally, all type 1 SMA infants older than 12 months require feeding support. In a natural history study, the median age of death or permanent ventilation for type 1 SMA infants was 13.5 months. In the FIREFISH study, 93 percent of infants were alive after 12 months of treatment and 85 percent were event-free. The median duration of treatment at the time of analysis was 15.2 months, with a median age of 20.7 months.

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