Publication: AAV2-hAADC (eladocagene exuparvovec) biodistribution and expression: Superiority of intraputaminal versus intracerebroventricular and intrathecal (lumbar) routes of administration

This research article, published in the International Journal of Toxicology, assesses alternative routes of human aromatic L-amino acid decarboxylase (hAADC) enzyme administration in monkeys by evaluating its biodistribution and expression

  • Learn about minimally invasive dosing options using intraputaminal, intracerebroventricular, and intrathecal administration
  • Compare how the routes for administration affect transgene levels in the central nervous system

Compton DR, DeMarco SJ, Yalamanchili P. Int J Toxicol. 2023;42(2):135–145

Eladocagene exuparvovec is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Eladocagene exuparvovec (UpstazaTM) is a gene therapy indicated for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype, currently licensed in the European Member States, Great Britain, Iceland, Liechtenstein, Northern Ireland and Norway.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any product.

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-2300229 | January 2024
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.