Sign in or register to access exclusive content on this site
REGISTER

ACMG 2025 oral presentation: Administration of eladocagene exuparvovec using the SmartFlow magnetic resonance-compatible ventricular cannula in pediatric patients with aromatic L-amino acid decarboxylase deficiency

This oral presentation, presented at ACMG 2025, assesses the pharmacodynamics of eladocagene exuparvovec using the SmartFlow magnetic resonance (MR)-compatible ventricular cannula in pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency

  • Understand the SmartFlow MR-compatible ventricular cannula for intraputaminal administration
  • Review the pharmacodynamics of eladocagene exuparvovec through cerebrospinal fluid homovanillic acid levels and putaminal-specific [18F] fluorodopa uptake in patients with AADC deficiency
  • Learn about the safety of bilateral intraputaminal infusion of eladocagene exuparvovec in pediatric patients

This poster was developed and funded by PTC Therapeutics for ACMG 2025.

Fast-track access

Don't have an NPI number? Switch to manual verification
MED-US-CORP-2600009 | September 2026

Register now:

This field is for validation purposes and should be left unchanged.
  • 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:
* indicates a required field
Back to NPI verification
MED-US-CORP-2600009 | September 2026
MED-US-AADC-2600005 | February 2026

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 website may include scientific information about investigational and unlicensed products not approved for use within your country. Please refer to local regulatory guidance for country-specific information

© 2026 PTC Therapeutics. All rights reserved | MED-US-CORP-2600001 | February 2026

This content is protected.

To view it please enter your email address or NPI number below:

Register here to access the content on the site

MED-US-CORP-2600009 | September 2026

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.