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 an investigational gene therapy product currently being developed by PTC Therapeutics for the treatment of patients with deficiency of a protein called AADC. Eladocagene exuparvovec does not currently have a marketing authorization for the treatment of AADC deficiency.
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MED-ALL-AADC-2200023 | March 2022
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