Posted by Michael Wonder on 20 Mar 2024
PTC Therapeutics provides key regulatory updates (March 2024)
19 March 2024 - BLA submitted to FDA for Upstaza for the treatment of AADC deficiency -
PTC Therapeutics today announced the submission of a BLA to the US FDA for Upstaza (eladocagene exuparvovec), a gene therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency.
