Translarna®

Active substance

ataluren

Holder

PTC Therapeutics France

Status

closed

Indication

Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, inambulatory patients aged 5 years and older for patients who have been treated with this medicinal product as part of the clinical trials (studies 019 and 020E) that are currently in the close-out process.

Public documents

Approbation

Information for the patient

Informed consent

Dernière mise à jour

14/04/2025

Last updated on