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.
AADC deficiency is an ultra-rare, inherited genetic disease which typically manifests within the first year of life. It is caused by changes in the gene that produces the AADC enzyme which is needed to produce certain substances vital for the normal functioning of the brain and nerves, including dopamine and serotonin. These substances are used by cells in the brain and the nervous system to send signals and are crucial for the development of motor functions…
