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.Eladocageneexuparvovec 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
MED-ALL-AADC-2100520 I February 2022
Sign in or register to access exclusive content on this site
Register here to access the content on the site MED-ALL-CORP-2200029 | December 2022
You are now leaving MEDhub, a website provided by PTC Therapeutics. This link will take you to a website to which our Privacy Statement and our Terms and Conditions do not apply.
We encourage you to read the Privacy Policy and the Terms and Conditions of every website you visit. PTC Therapeutics does not endorse and/or influence the content found on websites not owned/operated by PTC Therapeutics.
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.