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

Eladocagene Exuparvovec is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-PTC-AADC-2500006 | September 2025

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

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MED-ALL-AADC-2400021 | June 2025

Publication: Patient selection considerations for AADC deficiency gene therapy

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