CNS 2021 poster: Reductions in oculogyric crisis duration and frequency in children with aromatic L-amino acid decarboxylase (AADC) deficiency treated with eladocagene exuparvovec gene therapy: Results from 3 clinical trials

Change in the duration and frequency of oculogyric crises (OGC) 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 the treatment of AADC deficiency in children is presented
  • The efficacy of eladocagene exuparvovec on the following primary endpoints is investigated: Duration (hours/week) of OGC episodes; Frequency (episodes/week) of OGC episodes; Percentage of time spent experiencing OGC episodes
  • 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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