ESGCT 2021 Symposium: Pioneering in gene therapies: Sharing our experience with aromatic L-amino acid decarboxylase (AADC) deficiency
An overview of the etiology of AADC deficiency, the rationale behind gene therapy approaches, and latest clinical data are summarized in the PTC Therapeutics symposium at ESGCT 2021
Learn about the role of pathogenic variants in the DDC gene that result in a monoamine neurotransmitter deficiency
Recognize the key clinical signs and symptoms and current management options for AADC deficiency
Understand the theoretical principles of gene therapy for the treatment of AADC deficiency
Find out about the latest published data for an investigational gene therapy for the treatment of AADC deficiency
This symposium was developed and funded by PTC Therapeutics at ESGCT 2021. It was not part of the official program of ESGCT 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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MED-ALL-AADC-2200021 | March 2022
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