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PTC Therapeuticsptcbio.com · Low evidencePTC Therapeutics is a global biopharmaceutical company that discovers, develops, and commercializes clinically differentiated medicines for children and adults with serious diseases of high unmet need. The company focuses on rare conditions such as Duchenne muscular dystrophy, phenylketonuria, AADC deficiency, and Friedreich’s ataxia, and has achieved several firsts in approved treatments, including the first gene therapy directly infused into the brain.
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