Gene Therapy Shows Promise for AADC Deficiency But Requires Early Diagnosis, Study Says

Early clinical trials of a gene therapy for aromatic amino acid decarboxylase (AADC) deficiency have yielded encouraging results for its safety and efficacy, a study highlights. But researchers call attention to the importance of a timely diagnosis for treatment success, as only early interventions could fully prevent long-term…

#AANAM – AGIL-AADC Gene Therapy Improves Motor, Cognitive, and Speech Skills in AADC Children

Treatment with AGIL-AADC gene therapy induces sustained improvements in motor, cognitive, and language measures in children with aromatic L-amino acid decarboxylase (AADC) deficiency for up to five years, researchers report. Their work, “AGIL-AADC Gene Therapy Results in Sustained Improvements in Motor and Developmental Milestones Over 5 Years in…