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Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Child Neurological Society (CNS) Annual Meeting | CNS 2024 oral presentation: Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase deficiency

CNS 2024 oral presentation: Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase deficiency

This CNS 2024 oral presentation focuses on the efficacy and safety of eladocagene exuparvovec gene therapy for motor development in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

  • Learn more about the signs, symptoms and causes of AADC deficiency
  • Understand the impact of eladocagene exuparvovec gene therapy on motor development by assessing key milestones in pediatric patients
  • Review the safety of eladocagene exuparvovec in patients with AADC deficiency
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

This oral presentation was developed and funded by PTC Therapeutics for CNS 2024.

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