ISPMD 2022 symposium: The shifting treatment landscape for pediatric movement disorders: Investigational gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency

View our symposium presented at ISPMD 2022, held in Barcelona, Spain, which discusses the potential role of an investigational gene therapy treatment in the management of AADC deficiency

  • The unmet needs of patients with AADC deficiency, an underdiagnosed and underreported neurometabolic condition, are highlighted
  • Investigational gene therapy as a potential new treatment option for patients with AADC deficiency is discussed
  • Key practical details around the management of patients with AADC deficiency in clinical practice are shared

This is a symposium organized and funded by PTC Therapeutics at the 2022 7th ISPMD in Barcelona, Spain.

This symposium recording has been chapterized to enable ease of viewing.

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-2200015 | April 2022
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