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Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency

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
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MED-US-CORP-2500008 | November 2025

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

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MED-US-AADC-2600007 | February 2026

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