First therapy for the rare genetic disorder AADC deficiency

May 20, 2022

"EMA has recommended granting a marketing authorisation in the European Union (EU) for Upstaza (eladocagene exuparvovec), a therapy for the treatment of adult and paediatric patients with severe aromatic L-amino acid decarboxylase (AADC) deficiency with a genetically confirmed diagnosis." Read more on the EMA website:

First therapy to treat rare genetic nervous system disorder AADC deficiency | European Medicines Agency (