Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | The American Academy of Neurology (AAN) | AAN 2023 oral presentation: Eladocagene exuparvovec gene therapy improves motor development ​in patients with ​ aromatic L-amino acid decarboxylase deficiency

AAN 2023 oral presentation: Eladocagene exuparvovec gene therapy improves motor development ​in patients with ​ aromatic L-amino acid decarboxylase deficiency

This oral presentation at AAN 2023 discussed eladocagene exuparvovec, a gene therapy for patients with aromatic L-amino acid decarboxylase (AADC) deficiency, focusing on the efficacy and safety data from three clinical trials

  • Discover more about eladocagene exuparvovec as a gene therapy treatment for AADC deficiency, a rare genetic disorder
  • Explore key motor milestone development before and after treatment with eladocagene exuparvovec
  • Discover the change in oculogyric crisis episodes, weight, and rates of respiratory tract infections following treatment
  • Learn about the safety profile of eladocagene exuparvovec

▼ 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.

Eladocagene exuparvovec (UpstazaTM) is a gene therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype, currently licensed in the European Member States, Great Britain, Iceland, Liechtenstein, Northern Ireland and Norway.

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

This oral presentation was developed and funded by PTC Therapeutics for AAN 2023.

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