Home | Central Nervous System | Aromatic L-amino acid decarboxylase (AADC) deficiency | Congress activities | Child Neurological Society (CNS) Annual Meeting | CNS 2021 poster: Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

CNS 2021 poster: Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

Changes in motor scale scores 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 effects of eladocagene exuparvovec on motor development in children with AADC deficiency is investigated in a small group of children across 3 single-center trials
  • The assessments for measuring the achievement of motor milestones using the Peabody Developmental Motor Scale, 2nd edition (PDMS-2) and the Alberta Infant Motor Scale (AIMS), are detailed
  • 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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