Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | International Symposium on Paediatric Movement Disorders (ISPMD) | ISPMD 2022 symposium: The shifting treatment landscape for pediatric movement disorders: Investigational gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency

ISPMD 2022 symposium: The shifting treatment landscape for pediatric movement disorders: Investigational gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency

View our symposium presented at ISPMD 2022, held in Barcelona, Spain, which discusses the potential role of an investigational gene therapy treatment in the management of AADC deficiency

  • The unmet needs of patients with AADC deficiency, an underdiagnosed and underreported neurometabolic condition, are highlighted
  • Investigational gene therapy as a potential new treatment option for patients with AADC deficiency is discussed
  • Key practical details around the management of patients with AADC deficiency in clinical practice are shared

▼ 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 is a symposium organized and funded by PTC Therapeutics at the 2022 7th ISPMD in Barcelona, Spain.

This symposium recording has been chapterized to enable ease of viewing.

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MED-ALL-AADC-2200015 | December 2023
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