CNS 2021 Symposium: 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
The PTC Therapeutics symposium at CNS 2021 presents different clinical perspectives and experiences of an investigational gene therapy for the treatment of AADC deficiency
Understand the etiology of AADC deficiency and the rationale behind intraputaminal gene replacement therapy treatment
Recognize the key clinical signs and symptoms of AADC deficiency and the importance of an accurate and timely diagnosis
Find out the latest clinical trial and real-world clinical practice experience of an investigational gene replacement therapy for the treatment of AADC deficiency
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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