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A observational study evaluating efficacy and safety of PTC-AADC for 5 year in children with Aromatic L-Amino Acid Decarboxylase (AADC) deficiency

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Trial Profile

A observational study evaluating efficacy and safety of PTC-AADC for 5 year in children with Aromatic L-Amino Acid Decarboxylase (AADC) deficiency

Status: Completed
Phase of Trial: Phase IV

Latest Information Update: 11 Jun 2021

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At a glance

  • Drugs Eladocagene exuparvovec (Primary)
  • Indications Aromatic amino acid decarboxylase deficiency; Inborn error amino acid metabolic disorders
  • Focus Therapeutic Use

    • Most Recent Events

    • 11 Jun 2021 New trial record
    • 22 Apr 2021 Primary endpoint (Peabody Developmental Motor Scale) has been met. according to the results presented at the 73rd Annual Meeting of the American Academy of Neurology.
    • 22 Apr 2021 Results presented at the 73rd Annual Meeting of the American Academy of Neurology

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