Headquartered in Warren, NJ Fiscal year ends December NASDAQ: PTCT ptcbio.com
PTC Therapeutics is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for children and adults living with rare disorders. Its commercial portfolio includes Sephience (sepiapterin) for phenylketonuria, Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, and Emflaza (deflazacort) for Duchenne muscular dystrophy. PTC also earns royalty revenue from Roche's Evrysdi (risdiplam) for spinal muscular atrophy under a collaboration agreement. The company's pipeline spans Huntington's disease (votoplam, partnered with Novartis; PTC303), Friedreich's ataxia (vatiquinone), and inflammatory diseases (PTC612, an NLRP3 inhibitor), among other programs. PTC leverages its expertise in RNA biology and small molecule drug discovery to advance transformative therapies across rare disease indications. Updated
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Lee Jackson PTC Therapeutics is a global biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines for children and adults living with rare disorders.
The Wall Street consensus 12-month price target for PTCT is $97.94.
