PTC applies its expertise in RNA biology and drug development to pioneer novel oral treatments for patients living with serious and life-threatening conditions. Learn More.
Ataluren (PTC124®) is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. Learn about ataluren for genetic disorders
The ACT DMD trial, a Phase 3 confirmatory trial for ataluren for patients with nmDMD, is open for enrollment. Learn about the ACT DMD trial
PTC's current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology and infectious diseases. Learn about our therapeutic areas