First therapy to treat rare genetic disorder AADC deficiency
EMA has recommended granting a marketing authorization in the European Union (EU) for Upstaza (eladocagene exuparvovec), a therapy for the treatment of adult and pediatric patients with severe aromatic L-amino acid decarboxylase (AADC) deficiency with a genetically confirmed diagnosis.
Patients with AADC typically experience developmental delays, weak muscle tone and inability to control the movement of the limbs. AADC deficiency is a long-term, debilitating and life-threatening condition because it can lead to multiple organ failures. Patients also experience intellectual disability, show irritability and are at risk of death in the first decade of life.

