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
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.Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.
MED-ALL-PTC-AADC-2500006 | September 2025
This poster was developed and funded by PTC Therapeutics for ACMG 2025.
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