Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Child Neurological Society (CNS) Annual Meeting | CNS 2022 Symposium: A Spotlight on the Management of AADC Deficiency: Experience With Investigational Intraputaminal Gene Replacement Therapy

CNS 2022 Symposium: A Spotlight on the Management of AADC Deficiency: Experience With Investigational Intraputaminal Gene Replacement Therapy

This symposium, presented at CNS 2022, provides an overview of aromatic L-amino acid decarboxylase (AADC) deficiency management, with a focus on an investigational intraputaminal gene replacement therapy

  • Discover AADC deficiency: A rare neurotransmitter disorder
  • Explore the real-world clinical experience with gene therapy for AADC deficiency
  • Review the delivery method of gene therapy for AADC deficiency
  • Listen to caregiver insights into the disease burden of this rare disorder

▼ 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 at pharmacovigilance@ptcbio.com

Eladocagene exuparvovec (Upstaza) is a gene therapy product 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 and Norway. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for eladocagene exuparvovec is available here. Please refer to your local country guidance for more information.

This symposium was developed and funded by PTC Therapeutics for CNS 2022. It is not part of the official program at CNS 2022.

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MED-ALL-AADC-2200293 | December 2022
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