Abstract
FIREFISH (NCT02913482) is an ongoing, multicentre, open-label study of risdiplam in infants aged 1–7 months with Type 1 spinal muscular atrophy (SMA) and two SMN2 gene copies. FIREFISH aims to determine the efficacy and safety of risdiplam, an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier.FIREFISH Part 1 (n=21) assesses the safety, tolerability, pharmacokinetics and pharmacodynamics of different risdiplam dose levels. In FIREFISH Part 1 there have been no drug-related safety findings leading to withdrawal from the study following ≤30 (median 19) months of treatment (data-cut: 2nd July 2019). The confirmatory Part 2 (n=41) investigates the efficacy of risdiplam at the dose selected in Part 1. The primary efficacy endpoint is the proportion of infants sitting without support after 12 months on treatment, as assessed by Item 22 of the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development, third edition.Here we report efficacy and safety data from the confirmatory Part 2 of the FIREFISH study in participants who have received treatment with risdiplam for a minimum of 12 months. FIREFISH Part 2 will provide important data on the efficacy and safety of risdiplam in infants with Type 1 SMA.g.baranello@ucl.ac.uk
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