
Duchenne can be caused by a number of genetic mutations (changes). Ataluren (Translarna®) is for use in patients with “nonsense mutations” in the dystrophin gene, which prematurely stop the production of a normal dystrophin protein and lead to a shortened and nonfunctional dystrophin protein. Translarna works in these patients by enabling the protein-making apparatus in cells to move past the nonsense mutation, allowing the cells to produce a functional dystrophin protein.
In August 2014 Translarna received marketing authorization in the European Union for the treatment of nonsense mutation Duchenne in ambulatory patients aged five years and older, representing the first-ever treatment approved for the underlying cause of the disease.
PTC is expanding commercial access to Translarna across Europe, the Middle East, Latin America, and Asia Pacific.
PTC has engaged in dialogue with the US Food and Drug Administration regarding a path forward to bring Translarna to patients in the US.
A Phase 3 study is currently actively recruiting around the world. Two trials to study dystrophin levels in nonsense mutation patients (both Translarna-treated and untreated) also began recruiting in 2019.
This program is sponsored by PTC Therapeutics.
JULY 2020 | PTC Therapeutics Presents at the PPMD 2020 Virtual Annual ConferencePre-recorded content for PPMD's 2020 Virtual Annual Conference On-Demand Library |
JUNE 2019 | PTC Therapeutics Presents at the PPMD 2019 Annual Conference |
JANUARY 2019 | Webinar: Translarna® (ataluren) Development UpdateOn January 16, 2019, PTC Therapeutics joined Parent Project Muscular Dystrophy for a webinar update to provide the community with an overview of the Translarna development program with a focus on clinical trials that are recruiting. |
JUNE 2018 | PTC Therapeutics Presents at the PPMD 2018 Annual Conference |
AUG 2016 | Webinar: PTC Therapeutics Provides Regulatory Update on TranslarnaOn August 1, 2016, PPMD hosted a webinar with PTC Therapeutics to discuss the latest news regarding Translarna. |