PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy

First-ever FDA approval for gene therapy directly administered to the brain -- Priority review voucher granted -- Broad label including children and adults -- PTC pioneers new approach for CNS drug delivery -WARREN, N.J., Nov. 13, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly admini ...