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 three 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 an investigational gene therapy product currently being developed by PTC Therapeutics for the treatment of patients with deficiency of a protein called AADC. Eladocagene exuparvovec does not currently have a marketing authorization for the treatment of AADC deficiency.

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US-AADC-0496 | June 2022
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