A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) In Patients with Nonsense Mutation Dystrophinopathy
Phase of Trial: Phase III
Latest Information Update: 30 Sep 2017
At a glance
- Drugs Ataluren (Primary)
- Indications Duchenne muscular dystrophy; Muscular dystrophies
- Focus Registrational; Therapeutic Use
- Acronyms ACT DMD
- Sponsors PTC Therapeutics
- 18 Jul 2017 According to a PTC Therapeutics media release, results from this trial published in The Lancet.
- 17 Jul 2017 Results assessing the efficacy and safety of ataluren in ambulatory boys with nonsense mutation Duchenne muscular dystrophy (DMD) published in The Lancet.
- 06 Jun 2017 According to a PTC Therapeutics media release, the U.S. Food and Drug Administration (FDA) has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna).
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Table of Contents
- At a glance
- Trial Overview
- Trial Details
- Trial Centres
- Trial History