APS 2021 Symposium: Innovating in rare neurometabolic disorders: A focus on AADC deficiency
View the key takeaways from the APS 2021 Symposium, sponsored by PTC Therapeutics, which discusses the characteristic features, diagnosis, and innovative treatments in development for aromatic L-amino acid decarboxylase (AADC) deficiency
Learn about the core diagnostic tests recommended by international consensus guidelines for diagnosis of AADC deficiency
Understand the key signs and symptoms for AADC deficiency to aid early identification of this rare disease in clinical practice
Hear about the potential role of gene therapy treatments in development for patients with AADC deficiency
This symposium was developed and funded by PTC Therapeutics at the 34th APS annual conference, held in Kassel, Germany. It was not part of the official program of APS 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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