Publication: Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency
This research article, published in Molecular Therapy, presents the results of long-term follow-up from the 3 eladocagene exuparvovec gene therapy trials for aromatic L-amino acid decarboxylase (AADC) deficiency
Understand the unmet needs of currently available treatment options, and the rationale of a gene therapy approach in the treatment of AADC deficiency
Review the long-term (>5 years) efficacy and safety outcomes of eladocagene exuparvovec treatment in patients with AADC deficiency are reviewed
Tai CH, et al. Mol Ther. 2022;30:509–518
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▼ 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 email@example.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-2200023 | October 2023
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