Publication: Gene therapy in the putamen for curing aromatic L-amino acid decarboxylase (AADC) deficiency and Parkinson’s disease

This article, published in EMBO Molecular Medicine, describes the role of the putamen as an established target site for gene therapy in treating AADC deficiency and Parkinson’s disease

  • The rationale for targeting the putamen for gene therapy for the treatment of AADC deficiency and Parkinson’s disease is discussed
  • AADC gene therapy and the proposed mechanism of action of intraputaminal AADC gene therapy is described
  • A broad overview of the efficacy and safety of intraputaminal AADC gene therapy evaluated in clinical trials in children with AADC deficiency is presented
  • Additional sites for AADC gene therapy in AADC deficiency and Parkinson’s disease are discussed
  • Recommendations for assessing different therapeutic strategies for treating AADC deficiency are proposed

Hwu P, Kiening K, Anselm I, et al. EMBO Mol Med. 2021;e14712

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

Eladocagene exuparvovec (UpstazaTM) is a gene therapy product 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 Union, Great Britain, Iceland, Liechtenstein, Norway, and Northern Ireland. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for eladocagene exuparvovec is available here. Please refer to your local country guidance for more information.

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MED-ALL-AADC-2100412 | March 2023
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