SSIEM 2022 poster: A two-part, international, real-world, observational registry of participants diagnosed with aromatic L-amino acid decarboxylase deficiency (AADC-d) with or without treatment with eladocagene exuparvovec

This poster, presented at SSIEM 2022, provides an overview of the AADCAware registry, which has been designed to improve understanding of AADC deficiency diagnosis, disease progression and standard of care

• Improve understanding of AADC deficiency progression, its impact on patients and the disease progression
• Learn more about current patient demographics and baseline characteristics of those enrolled in the AADCAware registry
• Review the percentage of patients diagnosed with AADC deficiency who achieve key motor development milestones

▼ 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 pharmacovigilance@ptcbio.com. Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any product.

Eladocagene exuparvovec (Upstaza) 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. 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 poster was developed and funded by PTC Therapeutics for SSIEM 2022. It is not part of the official program at SSIEM 2022.