Risdiplam: positive topline results reported in FIREFISH trial for spinal muscular atrophy

Written by Sharon Salt, Senior Editor

PTC Therapeutics (NJ, USA) has announced positive topline results from part II of the FIREFISH trial assessing the efficacy of risdiplam – an investigational oral mRNA splicing modifier – in infants with Type 1 spinal muscular atrophy (SMA). Risdiplam was reported to have met its primary endpoint of infants who are sitting without support after 12 months of treatment. The drug was also well tolerated and no treatment-related safety findings leading to withdrawal were observed. FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part I of the trial was a dose-finding study that was...

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