Ataluren (Translarna®)

Ataluren (Translarna®)


Phase |||

Therapeutic Approach

Restoring or Replacing Dystrophin

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.


This program is sponsored by PTC Therapeutics.

Related Studies

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Study of Ataluren in Previously Treated Participants

Media Library


Webinar: A Conversation with PTC Therapeutics (December 2022)

PTC Therapeutics joined PPMD for a community webinar on December 9, 2022 for a discussion regarding PTC’s history with the community and plans for the future, including activities to bring ataluren (Translarna) to US patients with nonsense mutations.
JULY 2020

PTC Therapeutics Presents at the PPMD 2020 Virtual Annual Conference

Pre-recorded content for PPMD's 2020 Virtual Annual Conference On-Demand Library
JUNE 2019

PTC Therapeutics Presents at the PPMD 2019 Annual Conference


Webinar: Translarna® (ataluren) Development Update

On 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 Translarna

On August 1, 2016, PPMD hosted a webinar with PTC Therapeutics to discuss the latest news regarding Translarna.

Join Our Mailing List