Patient selection considerations for AADC deficiency gene therapy

Patient selection considerations for AADC deficiency gene therapy

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...

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Main Authors: Agathe Roubertie, Irina Anselm, Bruria Ben‐Zeev, Wuh‐Liang Hwu, Ashutosh Kumar, Berrin Monteleone, Shin‐ichi Muramatsu, Vincenzo Leuzzi, Salvador Ibáñez, Scellig Stone, Phillip L. Pearl
Format: Article
Language:English
Published: Wiley 2024-03-01
Series:Annals of the Child Neurology Society
Subjects:
AADC deficiency
gene therapy
rare disease
Online Access:https://doi.org/10.1002/cns3.20052
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