PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines for the treatment of rare disorders. The company offers Translarna (ataluren) and Emflaza (deflazacort) for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients. It is developing Translarna, which is in Phase 2 clinical study for the treatment of nonsense mutation aniridia and nonsense mutation Dravet syndrome/CDKL5; and RG7916 and RO7034067 for the treatment of patients with spinal muscular atrophy, as well as PTC596 and PTC299, a small molecule dihydrooratate dehydrogenase (DHODH) inhibitor that inhibits de novo pyrimidine nucleotide synthesis, which is in Phase 1 clinical development stage to treat cancer patients. The company is also developing gene therapy product candidate that include PTC-AADC for the treatment of Aromatic L-amino acid decarboxylase deficiency. It has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., and the Spinal Muscular Atrophy Foundation to develop and commercialize compounds identified under its spinal muscular atrophy sponsored research program; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey.
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