Brain-Injected Gene Therapy for AADC Deficiency Approved

A pioneering FDA-approved treatment offers new hope for children with this rare genetic disorder.

Topline

The FDA has granted fast-track approval for Kebilidi, the first brain-injected gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency. This therapy restores dopamine production, potentially improving movement-related symptoms and quality of life in affected children and adults.

Study Details

Aromatic L-amino acid decarboxylase …