Publication: Symptoms and impact of aromatic L-amino acid decarboxylase (AADC) deficiency: A qualitative study and the development of a patient-centred conceptual model

This original study article, published in the Current Medical Research and Opinion journal outlines the first qualitative study to understand the symptoms of AADC deficiency and its impact on individuals’ health-related quality of life

  • Gain an insight into the symptoms, impact, and challenges experienced by individuals with AADC deficiency from the perspective of caregivers
  • Understand the range of symptoms and functional impairments experienced by individuals with AADC deficiency, and the impact these have on their daily life and health-related quality of life
  • Learn about potential approaches to treatment that may affect function and symptoms and therefore improve the daily lives and health-related quality of life of these individuals

Williams K, et al. Curr Med Res Opin. 2021;37:1353–1361

Once registered, you will be able to view the abstract and be provided the DOI and PubMed links for this publication.
Register now to unlock the content

Register now to access the content on this page

Already registered?
If not, register below

Registration
  • 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:

    *
    *
  • 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 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.
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 unsubscribe from updates at any time when receiving an email from PTC.

MED-ALL-AADC-2200023 | October 2023
Sign in or register to access exclusive content on this site 
  REGISTER

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 content is protected. To view it please enter your email address below:

Register here to access the content on the site
MED-ALL-CORP-2200029 | October 2023

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.