PTC Therapeutics, Inc. (NASDAQ: PTCT) today presented a new analysis of five-year results that shows its novel gene therapy, PTC-AADC, leads to profound improvements in children with aromatic L-Amino acid decarboxylase (AADC) deficiency, a previously intractable, fatal and devastating rare disorder of the central nervous system
September 29, 2021
· 7 min read