Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Arbeitsgemeinschaft für Pädiatrische Stoffwechselstörungen (APS) | APS 2021 infographic: Innovating in rare neurometabolic disorders: A focus on AADC deficiency

APS 2021 infographic: Innovating in rare neurometabolic disorders: A focus on AADC deficiency

View the infographic distributed by PTC Therapeutics at the APS 2021 Symposium, which introduces aromatic L-amino acid decarboxylase (AADC) deficiency, outlines key diagnostic steps in clinical practice, and discusses gene therapies in development

  • Recognize the key signs and symptoms for AADC deficiency to aid early identification of this rare disease in clinical practice
  • Understand the core diagnostic tests recommended by international consensus guidelines for diagnosis
  • Find out about the potential role of gene therapy treatments in development for patients with AADC deficiency

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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