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.
Register now to unlock the content
Register now to access the content on this page
Already registered?
If not, register below
MED-ALL-AADC-2200021 | March 2022
Sign in or register to access exclusive content on this site
Register here to access the content on the site MED-ALL-CORP-2200029 | December 2022
You are now leaving MEDhub, a website provided by PTC Therapeutics. This link will take you to a website to which our Privacy Statement and our Terms and Conditions do not apply.
We encourage you to read the Privacy Policy and the Terms and Conditions of every website you visit. PTC Therapeutics does not endorse and/or influence the content found on websites not owned/operated by PTC Therapeutics.
The content you are trying to access is not currently available.
We will be updating the MEDhub site regularly to provide you with up-to-date, insightful expert-led content. To be notified when new additions become available, register now.