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 Therapeutics at pharmacovigilance@ptcbio.com.

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

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