October 5, 2017
A gene therapy for the rare childhood condition AADC deficiency is moving closer to market, years ahead of schedule, thanks to a collaboration between researchers from NCATS Therapeutics for Rare and Neglected Diseases program and Agilis Biotherapeutics, Inc., of Cambridge, Massachusetts.
AADC deficiency is a disease that affects the production of signals that allow cells in the nervous system to communicate with each other. These signals are called neurotransmitters. Children affected by this disease often have trouble coordinating movements, especially of the head, face, and neck. Other symptoms of the condition include problems controlling blood pressure, heart rate, and body temperature.
Aromatic L-amino acid decarboxylase deficiency