Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | International Child Neurology Congress (ICNC) | ICNC 2022 symposium: New paradigm in AADC deficiency: Diagnosis and management

ICNC 2022 symposium: New paradigm in AADC deficiency: Diagnosis and management

This symposium, presented at ICNC 2022, provides an overview of the real-world diagnosis and management experience of aromatic L-amino acid decarboxylase (AADC) deficiency

  • Professor Fatih Ezgü provides an introduction into the practical considerations of diagnosis and management
  • Learn about treatment advances in AADC deficiency with Professor Karl Kiening
  • Gain insights into the caregiver’s diagnostic journey and quality of life with Richard and Judith Poulin

▼ 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 (UpstazaTM) 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 Union, 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 ICNC 2022.

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