Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Publications | Publication: AAV2-hAADC (eladocagene exuparvovec) biodistribution and expression: Superiority of intraputaminal versus intracerebroventricular and intrathecal (lumbar) routes of administration

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.

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MED-ALL-AADC-2300229 | January 2024
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