CNS 2021 infographic: Advancing the care of a pediatric neurotransmitter disorder: Experience with an investigational intraputaminal gene replacement therapy for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency
An overview of 3 clinical trials of an investigational gene therapy for the treatment of AADC is provided in this infographic, distributed at the PTC Therapeutics symposium at CNS 2021
Understand the rationale behind intraputaminal gene replacement therapy for the treatment of AADC deficiency
Learn about the clinical trial experience of an investigational gene replacement therapy for the treatment of AADC deficiency from 3 studies
This symposium was developed and funded by PTC Therapeutics at CNS 2021. It was not part of the official program of CNS 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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