Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Society for the Study of Inborn Errors of Metabolism (SSIEM) | SSIEM 2023 symposium: AADC deficiency: Advances in diagnosis and treatment

SSIEM 2023 symposium: AADC deficiency: Advances in diagnosis and treatment

This symposium, presented at SSIEM 2023, provides an overview of the identification and diagnosis of patients with aromatic L-amino acid decarboxylase (AADC) deficiency. It also focuses on the potential of gene therapy as a treatment option

  • Review current and emerging approaches for identifying and diagnosing patients with AADC deficiency
  • Explore eladocagene exuparvovec, an intraputaminal gene therapy, as a treatment option for AADC deficiency
  • Discover the clinical safety and efficacy data of eladocagene exuparvovec

▼ 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 pharmacovigilance@ptcbio.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.

This symposium was developed and funded by PTC Therapeutics for SSIEM 2023.

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MED-ALL-AADC-2300225 | October 2023
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