Notable examples include the AAV2-based medication Upstaza® (INN: Eladocagene exuparvovec) for aromatic L-amino acid decarboxylase (AADC) deficiency, sanctioned by the European Medicines Agency (EMA) in 2022, and Luxturna® (INN: voretigene neparvovec) for RPE65 mutation-associated retinal dystrophy, approved by FDA in 2017. The gene discussed is DDC; the disease is hyperinsulinemic hypoglycemia, familial, 4.