ESGCT 2022 symposium: Pioneering gene therapy delivery for patients with rare diseases: The AADC deficiency story
This symposium, presented at ESGCT 2022, provides an overview of gene therapy as a strategy for the management of AADC deficiency
Learn more about gene therapy as a therapeutic strategy for the management of AADC deficiency
Understand more about the surgical procedure of intraputaminal gene therapy
Discover key clinical outcomes from real-world experience of AADC deficiency treatment
▼ 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 firstname.lastname@example.org
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.
This symposium was developed and funded by PTC Therapeutics for ESGCT 2022.
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MED-ALL-AADC-2200257 | April 2023
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