Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | European Society of Gene and Cell Therapy (ESGCT) | ESGCT 2022 infographic: Pioneering gene therapy delivery for patients with rare diseases: The AADC deficiency story

ESGCT 2022 infographic: Pioneering gene therapy delivery for patients with rare diseases: The AADC deficiency story

This infographic, distributed during PTC Therapeutics symposium at ESGCT 2022, provides an overview of gene therapy as a strategy for the management of AADC deficiency

  • Learn more about gene therapy as a therapeutic strategy for the management of AADC deficiency
  • Understand more about the surgical procedure of intraputaminal gene therapy
  • Discover key clinical outcomes from real-world experience of AADC deficiency treatment

Eladocagene exuparvovec is currently an investigational gene therapy product in the US. In the European Union Member States, Iceland, Norway, Lichtenstein, Northern Ireland, and Great Britain, eladocagene exuparvovec is indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of aromatic L-amino acid decarboxylase (AADC) deficiency with a severe phenotype.

This infographic was developed and funded by PTC Therapeutics for ESGCT 2022.

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US-AADC-0538 | September 2023
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