Sign in or register to access exclusive content on this site
REGISTER

Home | Central nervous system | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up

Publication: Gene therapy for aromatic L-amino acid decarboxylase deficiency: Requirements for safe application and knowledge-generating follow-up

This original article, published in JIMD, explores the requirements and recommendations for patients who receive gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency

  • Learn more about AADC deficiency, a rare genetic disorder
  • Evaluate gene therapy as a treatment option for AADC deficiency
  • Review the consensus recommendations for intraputaminal gene therapy preparation, delivery, and management, including postoperative care
Always consult the prescribing information before prescribing any products

MED-US-CORP-2500008 | November 2025

Roubertie A, et al. J Inherit Metab Dis. 2023. Epub ahead of print

Once registered, you will be able to view the abstract and be provided with the DOI links for this publication.

Fast-track access

Don't have an NPI number? Switch to manual verification
MED-US-CORP-2600009 | September 2026

Register now:

This field is for validation purposes and should be left unchanged.
  • This Website is intended only for healthcare professionals (“Website“), is operated by PTC Therapeutics. (together with its affiliates “PTC”). In order to access the restricted content on this Website:
    Marketing permissions - 1(Required)
    Marketing permissions - 2(Required)
    Keep me updated on the latest MEDhub content available*(Required)
* indicates a required field
Back to NPI verification

PTC will delete immediately the information provided if we cannot confirm you are healthcare professional. Otherwise, in case of successful verification, PTC will retain the information provided for a period of two (2) years following your registration (i) only for the purposes of providing you access to this Website; and/or (ii) in case you signed up to receive PTC updates.

Providing your personal data and your consent is voluntary, and you have the right to withdraw your consent at any time, without giving any reasons. Please consult our Privacy Statement for more information on the processing of your personal data: https://ptcprivacy.com/. For any enquiry relating to the processing of your personal data or if you want to exercise your privacy related rights or withdraw your consent, you can contact PTC’s Privacy Office at: Dataprivacy@ptcbio.com.

You can opt-out from PTC updates at any time by clicking the 'unsubscribe' link at the bottom of any email.

MED-US-CORP-2600009 | September 2026
MED-US-AADC-2600010 | February 2026

Welcome to MEDhub

A website for healthcare professionals, provided by PTC Therapeutics

This educational website provides you with the latest scientific data and expert insights on the diagnosis and management of a range of rare diseases across the neuromuscular system, central nervous system, and other areas

This website may include scientific information about investigational and unlicensed products not approved for use within your country. Please refer to local regulatory guidance for country-specific information

© 2026 PTC Therapeutics. All rights reserved | MED-US-CORP-2600001 | February 2026

This content is protected.

To view it please enter your email address or NPI number below:

Register here to access the content on the site

MED-US-CORP-2600009 | September 2026

COMING SOON

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.