Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | International Symposium on Paediatric Movement Disorders (ISPMD) | ISPMD 2022 poster: Early clinical improvements following treatment with eladocagene exuparvovec in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

ISPMD 2022 poster: Early clinical improvements following treatment with eladocagene exuparvovec in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

The key findings of a post hoc analysis of the clinical trial results for an investigational gene therapy are discussed in our poster from ISPMD 2022, held in Barcelona, Spain

  • The clinical outcomes from 3 open-label clinical trials evaluating the efficacy of a gene therapy for AADC deficiency are discussed
  • Results from 28 patients who had intraputaminal injections of eladocagene exuparvovec, at baseline and at up to 12 months’ follow-up, are presented
  • Clinical outcomes, including gross motor milestone development and physical and neurological functions, are reported

▼ 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-2200015 | December 2023
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