Ptc Therapeutics Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of medicines for patients living with rare disorders, particularly orphan diseases aff... Ptc Therapeutics Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of medicines for patients living with rare disorders, particularly orphan diseases affecting children and adults. The company specializes in orally administered small molecule drugs and gene therapies that target post-transcriptional control mechanisms to regulate gene expression. Its approved products include Translarna for nonsense mutation Duchenne muscular dystrophy, Emflaza for Duchenne muscular dystrophy, Upstaza for aromatic L-amino acid decarboxylase deficiency, a central nervous system disorder, and commercialization rights for WAYLIVRA in Latin America. PTC also collaborates on Evrysdi for spinal muscular atrophy. The company's pipeline features investigational therapies such as sepiapterin for phenylketonuria and vatiquinone for Friedreich's ataxia, alongside platforms addressing inflammation, ferroptosis, and Huntington's disease. As a patient-centric organization headquartered in South Plainfield, New Jersey since 1998, Ptc Therapeutics Inc. leverages scientific expertise and global commercial infrastructure to deliver transformative treatments for diseases with high unmet medical needs.Show lessShow more

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