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
ACT CF, a Phase 3 confirmatory trial for ataluren for patients with nmCF, is open for enrollment. Learn about the ACT CF 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