PTC Therapeutics is a biopharmaceutical company focused on the discovery and development of innovative therapies for the treatment of rare diseases and cancer
The company specializes in the development of small molecules and gene therapies that target underlying genetic causes of diseases. PTC Therapeutics aims to advance its proprietary drug candidates through clinical trials, leveraging its unique expertise in RNA biology and translational medicine. By utilizing cutting-edge technology and a collaborative approach, the company strives to bring transformative treatments to patients with unmet medical needs.
PTC Therapeutics halts Utreloxastat development after ALS trial disappoints. Analysts highlight promising pipeline, including Huntington's and PKU programs.
PTC Therapeutics released interim results from its Phase 2 PIVOT-HD study on PTC518 for Huntington's disease. The FDA has lifted the partial clinical hold on the program.
PTC Therapeutics announced that the European Commission has asked the CHMP to re-evaluate its negative opinion on the renewal of Translarna's conditional marketing authorization, keeping it available in Europe.
