Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Patient selection considerations for AADC deficiency gene therapy

Publication: Patient selection considerations for AADC deficiency gene therapy

This review article, published in Annals of the Child Neurology Society, presents the considerations for patient selection of disease‐modifying gene therapy for aromatic L‐amino acid decarboxylase (AADC) deficiency

Learn more about AADC deficiency, a rare, severe, genetic neurological disorder that results in a combined deficiency of several key neurotransmitters
Review the pivotal clinical trial data for eladocagene exuparvovec, a gene therapy for AADC deficiency
Understand the considerations for selecting patients with AADC deficiency for gene therapy

Always consult the prescribing information before prescribing any products

MED-US-CORP-2500008 | November 2025

Roubertie A, Anselm I, Ben-Zeev B, et al. Ann Child Neurol Soc. 2024;2(1):53–59

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MED-US-AADC-2600011 | February 2026

Publication: Patient selection considerations for AADC deficiency gene therapy

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