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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

GEMS^™ (Gene Expression Modulation by Small-Molecules) is a novel and proprietary screening technology for the identification of small molecules. Learn about the GEMS^™ advantage

Rare Disease Day is an international advocacy day to bring widespread recognition of rare diseases as a global health challenge. Learn about Rare Disease Day

Our Pipeline

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

12/22/2011PTC Therapeutics' Antibacterial Program Receives $5 Million Award From The Wellcome Trust

11/29/2011Roche Signs Agreement With PTC Therapeutics To Advance Treatment For Spinal Muscular Atrophy (sma)

11/7/2011PTC Therapeutics Receives Grand Challenges Explorations Funding

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