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Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up

Publication: Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up

This original article, published in JIMD, explores the requirements and recommendations for patients who receive gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency

  • Learn more about AADC deficiency, a rare genetic disorder
  • Evaluate gene therapy as a treatment option for AADC deficiency
  • Review the consensus recommendations for intraputaminal gene therapy preparation, delivery, and management, including postoperative care
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, et al. J Inherit Metab Dis. 2023. Epub ahead of print

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MED-ALL-AADC-2300229 | June 2025

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