A Phase 3 Randomized Double Blind Placebo Controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) and Open Label Extension
Phase of Trial: Phase III
Latest Information Update: 27 Feb 2018
At a glance
- Drugs Ataluren (Primary)
- Indications Duchenne muscular dystrophy
- Focus Therapeutic Use
- Sponsors PTC Therapeutics
- 20 Feb 2018 According to a PTC Therapeutics media release, the letter from US FDA adds that PTC Study 041 could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework for ataluren.
- 11 Jul 2017 Status changed from not yet recruiting to recruiting.
- 05 Jun 2017 Status changed from planning to not yet recruiting.