Publication: Pharmacodynamics, efficacy, and safety of intraputaminal eladocagene exuparvovec administered to pediatric patients with aromatic L-amino acid decarboxylase deficiency using an MR-compatible cannula: 48 weeks of follow-up
This research article explores the administration of eladocagene exuparvovec using the SmartFlow magnetic resonance (MR)-compatible ventricular cannular in pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency
- Review the pathophysiology of AADC deficiency, a rare neurotransmitter-related disease
- Learn about the SmartFlow MR-compatible ventricular cannular for intraputaminal administration
- Assess the pharmacodynamics, efficacy and safety of eladocagene exuparvovec using the SmartFlow MR-compatible ventricular cannular in this population
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
Curry DJ, Pearl PL, Stone SSD, et al. J Inherit Metab Dis. 2026;49(2);e70151.
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