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PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders. The company's commercial product is Emflaza (deflazacort) used for the treatment of Duchenne muscular dystrophy in ambulatory patients aged 5 years and older. Its lead product candidate is Translarna that is completed Phase III clinical trials for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients; which has completed Phase II/III clinical trials for the treatment of cystic fibrosis; and that is under preclinical development stage for the treatment of myotonic dystrophy type 1.
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PTCT
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🇺🇸 United States