CNS 2021 poster: Eladocagene exuparvovec improves body weight and reduces respiratory infections in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

Changes in body weight and rate of respiratory infections in children with AADC deficiency following treatment with eladocagene exuparvovec, an investigational gene therapy, are presented in this poster from the 2021 CNS Annual Meeting

  • The role of intraputaminal infusion of eladocagene exuparvovec as a gene therapy for treating the symptoms of AADC deficiency in children is presented
  • The efficacy of eladocagene exuparvovec in increasing body weight (a positive indicator of AADC deficiency) is investigated in a small group of children across 3 single-center trials
  • The effects of eladocagene exuparvovec on minimizing respiratory infections and pneumonia were also assessed in this patient population
  • Treatment emergent adverse events with eladocagene exuparvovec are also described

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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MED-ALL-AADC-2100520 I February 2022
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