newsFirst gene therapy recommended for AADC23 March 2023 | By Catherine Eckford (European Pharmaceutical Review)NICE's final draft guidance has recommended the first and only gene therapy for children with aromatic L-amino acid decarboxylase (AADC) deficiency.
newsMHRA approves first gene therapy for infusion into the brain18 November 2022 | By Catherine Eckford (European Pharmaceutical Review)Upstaza™, approved for UK patients 18 months and over, is the first gene replacement therapy infused directly into the brain for the treatment of AADC deficiency.
