PTC THERAPEUTICS, INC.

PTC Therapeutics is a biopharmaceutical company focused on discovering, developing, and commercializing therapeutic medicines for rare genetic and neurological disorders in pediatric and adult populations. The company's marketed products address several distinct therapeutic areas. In muscular dystrophy, it offers Translarna and Emflaza for Duchenne muscular dystrophy treatment, and Evrysdi for spinal muscular atrophy across multiple age groups. For metabolic and neurological conditions, the company markets Upstaza (also branded as Kebilidi) for aromatic L-amino acid decarboxylase deficiency, a central nervous system disorder. Additional marketed medicines include Tegsedi and Waylivra for other rare disease indications. The company maintains an active development pipeline featuring candidates such as Sepiapterin for phenylketonuria and PTC518, a splicing platform program in collaboration with Novartis targeting Huntington's disease. Other pipeline assets address inflammation and ferroptosis pathways, including vatiquinone for Friedreich's ataxia.

PTC Therapeutics operates with approximately 991 full-time employees and maintains its headquarters in Warren, New Jersey. The company distributes products through third-party distributors and serves markets across the United States and internationally. It has established partnerships with major pharmaceutical companies including Novartis, Roche, and Akcea Therapeutics, alongside collaborations with research institutions and patient advocacy organizations such as the SMA Foundation.