Translating Science. Transforming Lives.
Our mission at PTC Therapeutics has always been to build an integrated biopharmaceutical company based on our expertise in RNA biology. We have focused on discovering and developing novel oral treatments for patients with serious and life-limiting disorders. By targeting the processes that modulate RNA biology and affect protein production, we bring an innovative approach to drug discovery. We are focused particularly on the development and commercialization of treatments for rare and neglected disorders.
Commitment to Patients.
We are committed to finding treatment options for patients living with life-threatening diseases. Our lead product candidate is ataluren for the treatment of patients with genetic disorders due to a nonsense mutation. Ataluren is in clinical development for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) and nonsense mutation cystic fibrosis (nmCF). Ataluren has been approved under the trade name Translarna™ in the European Union for ambulatory patients with nmDMD who are 5 years and older.