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

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

This infographic, distributed at the PTC Therapeutics symposium at ICIEM 2021, discusses key signs and symptoms, diagnostic tests, best practice management, and investigational gene therapy treatments in development for AADC deficiency

  • Learn about the etiology and clinical features of AADC deficiency, and how to differentiate from other neurotransmitter disorders
  • Understand the core diagnostic tests that should be used to confirm a diagnosis of AADC deficiency
  • Know what treatment options are currently available for AADC deficiency
  • Find out about potential investigational gene therapy treatments that are in development

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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