PTC Therapeutics is a biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. The company has the following products, Translarna? (ataluren) and Emflaza? (deflazacort), for the treatment of Duchenne muscular dystrophy, a rare, life threatening disorder. The company's gene therapy product candidates for rare monogenic diseases that affect the central nervous system (CNS) include PTC-AADC for the treatment of Aromatic L-Amino Acid Decarboxylase (AADC) deficiency, a rare CNS disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene.
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