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The website is organised and funded by PTC Therapeutics for healthcare professionals in Middle East and Africa
What is gene therapy?

Gene therapy aims to treat the genetic disorders by replacing or inactivating genes or introducing corrective genes into cells1

Find out more about gene therapy and the future on AADC deficiency management:

References: 1. American Society of Gene and Cell Therapy. Available at: https://patienteducation.asgct.org/gene-therapy-101/gene-therapybasics.Last accessed October 2021

AE-AADC-0267 | August 2022

Welcome to "About AADC MEA"

This website is provided for healthcare professionals residing in the Middle East and Africa.

This educational website provides information to support the early and accurate diagnosis of rare neurotransmitter disorders, including Aromatic L-amino Acid Decarboxylase (AADC) deficiency.