Publication: Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency

This research article, published in Molecular Therapy, presents the results of long-term follow-up from the 3 eladocagene exuparvovec gene therapy trials for aromatic L-amino acid decarboxylase (AADC) deficiency

Understand the unmet needs of currently available treatment options, and the rationale of a gene therapy approach in the treatment of AADC deficiency
Review the long-term (>5 years) efficacy and safety outcomes of eladocagene exuparvovec treatment in patients with AADC deficiency are reviewed

Tai CH, et al. Mol Ther. 2022;30:509–518

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Eladocagene exuparvovec is currently an investigational gene therapy product in the US. Eladocagene exuparvovec has been granted marketing authorization by the European Commission for the European Union Member States, Iceland, Norway, Northern Ireland, and Lichtenstein and granted marketing authorization by the UK MHRA for Great Britain.

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Publication: Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency

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