Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Society for Inherited Metabolic Disorders (SIMD) | SIMD 2022 symposium: Advancing the diagnosis and management of a rare neurometabolic disorder: A focus on AADC deficiency

SIMD 2022 symposium: Advancing the diagnosis and management of a rare neurometabolic disorder: A focus on AADC deficiency

This satellite symposium, presented at the 2022 SIMD Annual Meeting in Orlando, Florida, focuses on the diagnosis and management of aromatic L-amino acid decarboxylase (AADC) deficiency

  • Learn about the biochemical tests to diagnose AADC deficiency
  • Understand the variants in the DDC gene that are associated with AADC deficiency and ways to improve their interpretation
  • Find out about the latest 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 SIMD 2022. It was not part of the official program of SIMD 2022.

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-2200063 | July 2022
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