Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Society for the Study of Inborn Errors of Metabolism (SSIEM) | SSIEM 2021 Symposium: Rare disorders of monoamine metabolism: A focus on aromatic L-amino acid decarboxylase (AADC) deficiency

SSIEM 2021 Symposium: Rare disorders of monoamine metabolism: A focus on aromatic L-amino acid decarboxylase (AADC) deficiency

View the PTC Therapeutics symposium at SSIEM 2021, which discusses the key signs and symptoms, diagnostic tools, and management options in AADC deficiency

  • Learn about the genetic basis of rare monoamine metabolism disorders
  • Recognize the key clinical features of AADC deficiency to help distinguish the condition from other pediatric neurotransmitter disorders with similar symptoms, and understand the screening and core diagnostic tests that should be used to confirm an AADC deficiency diagnosis
  • Understand the current management options for the treatment of AADC deficiency
  • Find out about an investigational gene therapy approach for the treatment of AADC deficiency

This symposium was developed and funded by PTC Therapeutics at SSIEM 2021. It was not part of the official program of SSIEM 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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US-AADC-0496 | June 2022
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