Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | International Congress of Inborn Errors of Metabolism (ICIEM) | ICIEM 2021 Symposium: Rare neurometabolic disorders: A focus on aromatic L-amino acid decarboxylase (AADC) deficiency

ICIEM 2021 Symposium: Rare neurometabolic disorders: A focus on aromatic L-amino acid decarboxylase (AADC) deficiency

View the PTC Therapeutics symposium at ICIEM 2021, which reviews clinical and real-life experiences of AADC deficiency and provides an overview of current and potential future management options

  • Understand the etiology and clinical features of AADC deficiency, and how this differs from other neurotransmitter disorders
  • Learn from clinical cases that illustrate real-life presentation, key signs and symptoms, and steps to diagnosis
  • Management approaches for the treatment of AADC deficiency are outlined
  • Learn about the clinical trial experience of an investigational gene replacement therapy for the treatment of AADC deficiency

This symposium was developed and funded by PTC Therapeutics at ICIEM 2021. It was not part of the official program of ICIEM 2021.

Eladocagene exuparvovec is an investigational gene therapy product currently being developed by PTC Therapeutics for the treatment of patients with deficiency of a protein called AADC. Eladocagene exuparvovec does not currently have a marketing authorization for the treatment of AADC deficiency.

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MED-ALL-AADC-2200021 | March 2022
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