Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | European Society of Gene and Cell Therapy (ESGCT) | ESGCT 2023 symposium: Breaking down barriers: Bringing the benefits of gene therapies to patients

ESGCT 2023 symposium: Breaking down barriers: Bringing the benefits of gene therapies to patients

This symposium, presented at ESGCT 2023, provides an overview of the benefits of gene therapy for patients with aromatic L-amino acid decarboxylase (AADC) deficiency

  • Review what AADC deficiency is and common signs and symptoms
  • Learn more about the role of gene therapy as a therapeutic strategy for the management of AADC deficiency
  • Explore real-world case studies of children with AADC deficiency who have received gene therapy treatment
  • Discover PTC’s heritage in gene therapy and how a new treatment is developed

▼ 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 ESGCT 2023.

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MED-ALL-AADC-2300238 | December 2023
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