Europaediatrics 2021 Symposium: Recognising rare neurometabolic disorders – The journey starts with you
View key takeaways from the Europaediatrics 2021 Symposium, hosted by PTC Therapeutics, which reviews the signs and symptoms, referral pathway, and potential of gene therapy for the management of aromatic L-amino acid decarboxylase (AADC) deficiency
Understand the signs and symptoms of AADC deficiency and how to differentiate these from other neurotransmitter disorders
Learn about the role of the pediatric neurologist in referral pathways to drive early and accurate diagnosis of this rare disease
Find out more about the role of gene therapy as a potential treatment option for patients with AADC deficiency
This symposium was developed and funded by PTC Therapeutics at the 2021 Europaediatrics Congress, online and in Zagreb, Croatia. It was not part of the official program of Europaediatrics 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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