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
  • Explore gene therapy as a treatment option for AADC deficiency
  • Review the consensus recommendations for intraputaminal gene therapy preparation, delivery, and management, including postoperative care

Roubertie A, et al. J Inherit Metab Dis. 2023. Epub ahead of print

▼ 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.

Eladocagene exuparvovec (UpstazaTM) is a gene therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype, currently licensed in the European Member States, Great Britain, Iceland, Liechtenstein, Northern Ireland and Norway.

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

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