Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | International Symposium on Paediatric Movement Disorders (ISPMD) | ISPMD 2022 symposium: The shifting treatment landscape for pediatric movement disorders: Investigational gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency

ISPMD 2022 symposium: The shifting treatment landscape for pediatric movement disorders: Investigational gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency

View our symposium presented at ISPMD 2022, held in Barcelona, Spain, which discusses the potential role of an investigational gene therapy treatment in the management of AADC deficiency

  • The unmet needs of patients with AADC deficiency, an underdiagnosed and underreported neurometabolic condition, are highlighted
  • Investigational gene therapy as a potential new treatment option for patients with AADC deficiency is discussed
  • Key practical details around the management of patients with AADC deficiency in clinical practice are shared

This is a symposium organized and funded by PTC Therapeutics at the 2022 7th ISPMD in Barcelona, Spain.

This symposium recording has been chapterized to enable ease of viewing.

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

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 a 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.
For any inquiry 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.

US-AADC-0496 | June 2022
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
US-CORP-0330 | July 2022

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.