PTC Therapeutics(PTCT) Prnewswire·2025-03-28 13:00Core Viewpoint - The European Commission has decided not to renew the authorization of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy, although individual EU member states may still allow its use under specific provisions [1][2]. Company Overview - PTC Therapeutics, Inc. is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, leveraging scientific expertise and a global commercial infrastructure to maximize value for patients [5]. Product Information - Translarna (ataluren) is a protein restoration therapy aimed at enabling the formation of functional proteins in patients with genetic disorders caused by nonsense mutations, specifically targeting Duchenne muscular dystrophy [3]. - Translarna is licensed in multiple countries for treating nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged 2 years and older, while it remains an investigational new drug in the United States [3]. Disease Context - Duchenne muscular dystrophy is a rare and fatal genetic disorder primarily affecting males, leading to progressive muscle weakness and premature death due to heart and respiratory failure, with the absence of functional dystrophin protein being a critical factor [4].