Approved treatments
for AADC

Aromatic L-amino acid decarboxylase (AADC) deficiency treatment may include gene therapy, medications, and supportive therapies to help manage symptoms and improve quality of life. Gene therapy is the first disease-modifying treatment approved for AADC deficiency and is intended to address the underlying genetic cause of the disease. A treatment plan tailored to each person’s needs can help manage symptoms, support development, and improve daily functioning.

Off-label medications are often used to help manage symptoms and improve quality of life in people with AADC deficiency. Commonly used medications include vitamin B6, dopamine receptor agonists, and monoamine oxidase inhibitors, which work by increasing the production or effects of dopamine and other neurotransmitters affected by the disease.