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

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

This infographic, presented at ESGCT 2023, provides an overview of the signs and symptoms of aromatic L-amino acid decarboxylase (AADC) deficiency and the use of gene therapy for treating this rare disease

  • Learn more about the signs and symptoms of AADC deficiency
  • Understand the use of gene therapy as a treatment strategy for AADC deficiency
  • Explore real-world safety and efficacy data on the use of gene therapy

▼ 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.

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MED-ALL-AADC-2300240 | November 2023
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