Author Archives: Steve Bryson PhD

PTC-AADC Gene Therapy Safe, Showing Promise for Children

Using PTC-AADC gene therapy in children with aromatic l-amino acid decarboxylase (AADC) deficiency was found to be relatively safe and led to improvements in motor function, cognitive abilities, and breathing, a review of six clinical trials found. The therapy also showed benefits in feeding, mood, and sleep among…

Italian Expert Panel Issues Diagnostic, Treatment Guidelines

An Italian panel of experts recommends using vitamin B6, dopamine agonists, and monoamine oxidase inhibitors (MAOIs) as first-line therapies for the treatment of people with rare aromatic l-amino acid decarboxylase (AADC) deficiency. Further, the expert panel called for an across-the-board, multidisciplinary approach for follow-up management of patients…

Non-invasive MRI May Help Diagnose AADC Deficiency

Systematic analysis of non-invasive MRI brain scans of people with aromatic l-amino acid decarboxylase (AADC) deficiency identified disease-related changes in brain structure, which may help diagnose the disorder and related conditions, a study suggests. The study, “…