Patient selection considerations for AADC deficiency gene therapy

• Main Authors: Agathe Roubertie (Author), Irina Anselm (Author), Bruria Ben‐Zeev (Author), Wuh‐Liang Hwu (Author), Ashutosh Kumar (Author), Berrin Monteleone (Author), Shin‐ichi Muramatsu (Author), Vincenzo Leuzzi (Author), Salvador Ibáñez (Author), Scellig Stone (Author), Phillip L. Pearl (Author)
• Format: Book
• Published: Wiley, 2024-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Aromatic ʟ‐amino acid decarboxylase (AADC) deficiency is a rare, severe neurological disorder caused by pathogenic variants in the dopa decarboxylase (DDC) gene, resulting in a combined deficiency of monoamine neurotransmitters. Clinically, patients present with a range of dysfun...