Trial Profile

A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy

Status: Completed

Phase of Trial: Phase II

Latest Information Update: 09 Aug 2024

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At a glance

Drugs Risdiplam (Primary)
Indications Spinal muscular atrophy
Focus Adverse reactions; Pharmacodynamics; Pharmacokinetics; Registrational; Therapeutic Use
Acronyms FIREFISH
Sponsors Chugai Pharmaceutical; Roche

07 Jun 2024 According to Roche media release, data from this study presented at the Cure SMA Research & Clinical Care Meeting
07 Jun 2024 Results published in the Roche Media Release
01 Feb 2024 This trial has been Completed in Poland, According to European Clinical Trials Database record.

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