Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Society for the Study of Inborn Errors of Metabolism (SSIEM) | SSIEM 2022 symposium: Transformative science in action: Gene therapy for AADC deficiency

SSIEM 2022 symposium: Transformative science in action: Gene therapy for AADC deficiency

This symposium, from the 15th SSIEM annual congress in 2022, held in Freiburg, Germany, gives real-world insights into the management of aromatic L-amino acid decarboxylase (AADC) deficiency from a caregiver and clinician perspective

  • Explore long-term efficacy and safety data from AADC deficiency clinical trials assessing eladocagene exuparvovec
  • Learn about the caregiver burden associated with an AADC deficiency diagnosis
  • Discover real-world outcomes of treatment with eladocagene exuparvovec in patients with AADC deficiency

▼ 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

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 SSIEM 2022.

This is a recording of our live webinar. This webinar recording has been chapterized to enable ease of viewing.
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MED-ALL-AADC-2300061 | May 2023
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