PTC Therapeutics Inc (PTCT)

PTC Therapeutics, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing medicines for children and adults living with rare disorders in the United States and international markets. Its approved products include Translarna and Emflaza for Duchenne muscular dystrophy, Upstaza for AADC deficiency, Tegsedi and Waylivra for rare diseases, and Evrysdi for spinal muscular atrophy. The company also markets Upstaza under the brand name Kebilidi in the United States. In addition to its commercial portfolio, PTC Therapeutics is advancing several pipeline programs, including Sepiapterin for phenylketonuria, the PTC518 splicing platform for Huntington’s disease, and inflammation and ferroptosis programs such as vatiquinone for Friedreich’s ataxia. The company works with third-party distributors and has collaborations with Roche, the SMA Foundation, National Taiwan University, Akcea Therapeutics, Shiratori Pharmaceutical, and Novartis. PTC Therapeutics was incorporated in 1998 and is headquartered in Warren, New Jersey.

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