Publication: Intraputaminal gene delivery in two patients with aromatic L-amino acid decarboxylase deficiency
This case study published in Movement Disorders describes the outcomes of 2 patients with aromatic L-amino acid decarboxylase (AADC) deficiency treated with intraputaminal gene therapy
Learn more about intraputaminal gene therapy for AADC deficiency, a rare autosomal recessive disease
Review the key efficacy outcomes of eladocagene exuparvovec treatment in 2 patients over 10 years of age with severe forms of AADC deficiency
Discover the safety profile of eladocagene exuparvovec for these cases
François-Heude MC, et al. Mov DisordClin Pract. 2023. Online ahead of print.
▼ 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 at email@example.com
Eladocagene exuparvovec (UpstazaTM) is a gene therapy product 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 Union, Great Britain, Iceland, Liechtenstein and Norway. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for eladocagene exuparvovec is available here. Please refer to your local country guidance for more information.
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MED-ALL-AADC-2300018 | April 2023
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