PTC Therapeutics announced it will withdraw the ataluren NDA for nmDMD, citing FDA concerns regarding the evidence of the agent's effectiveness.

PTC Therapeutics, Inc. PTCT closed lower on Thursday following the company’s decision to withdraw its New Drug Application for Translarna after receiving feedback from the U.S. Food and Drug ...

The firm originally submitted a new drug application seeking approval of the drug in 2016, which was rejected, and, in 2024, resubmitted an application.

PTC Therapeutics withdrew its New Drug Application (NDA) resubmission for Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy (nmDMD) following feedback from FDA indicating that the ...

Researchers develop a single genome-editing strategy to treat multiple disorders caused by nonsense mutations, promising efficient and cost-effective therapies.

In a statement, PTC's chief executive, Matthew Klein, said the company had decided to pull the filing for Translarna (ataluren) as a treatment for nonsense mutation DMD after the FDA said it was ...

From infancy and usually for life, some families suffer from broken hair due to a congenital form of hair loss called monilethrix. Researchers at the University Hospital Bonn and the University of ...

Investing.com -- PTC Therapeutics (NASDAQ:PTCT) stock fell 4% in after-hours trading on Thursday after the company announced it had withdrawn its New Drug Application (NDA) resubmission for Translarna ...

From infancy and usually for life, some families suffer from broken hair due to a congenital form of hair loss called monilethrix. Researchers at have now identified causative mutations in another ...