ISPMD 2024 poster: Pharmacodynamics of eladocagene exuparvovec and safety of the SmartFlow magnetic resonance-compatible ventricular cannula for administering eladocagene exuparvovec in paediatric participants

This poster, presented at ISPMD 2024, 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
Discover the SmartFlow MR-compatible ventricular cannular for intraputaminal administration
Assess the pharmacodynamics and safety of eladocagene exuparvovec using the SmartFlow MR-compatible ventricular cannular in pediatric patients with AADC deficiency

▼ 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 (Upstaza^TM) 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.

This poster was developed and funded by PTC Therapeutics for ISPMD 2024.

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ISPMD 2024 poster: Pharmacodynamics of eladocagene exuparvovec and safety of the SmartFlow magnetic resonance-compatible ventricular cannula for administering eladocagene exuparvovec in paediatric participants

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