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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
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.

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

MED-ALL-PTC-AADC-2500006 | September 2025

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

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MED-ALL-AADC-2200023 | June 2025

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