Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Child Neurological Society (CNS) Annual Meeting | CNS 2021 poster: Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

CNS 2021 poster: Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

Changes in motor scale scores in children with AADC deficiency following treatment with eladocagene exuparvovec, an investigational gene therapy, are presented in this poster from the 2021 CNS Annual Meeting 

  • The role of intraputaminal infusion of eladocagene exuparvovec as a gene therapy for the treatment of AADC deficiency in children is presented
  • The effects of eladocagene exuparvovec on motor development in children with AADC deficiency is investigated in a small group of children across 3 single-center trials
  • The assessments for measuring the achievement of motor milestones using the Peabody Developmental Motor Scale, 2nd edition (PDMS-2) and the Alberta Infant Motor Scale (AIMS), are detailed
  • Treatment emergent adverse events with eladocagene exuparvovec are also described

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 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-2100520 I February 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
GL-CORP-0207 | June 2021

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.