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Prospective pivotal study of AADC gene therapy (AAV-hAADC gene therapy) in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

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Trial Profile

Prospective pivotal study of AADC gene therapy (AAV-hAADC gene therapy) in patients with aromatic L-amino acid decarboxylase (AADC) deficiency

Status: Planning
Phase of Trial: Phase III

Latest Information Update: 22 Nov 2018

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At a glance

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

    • Most Recent Events

    • 02 Mar 2016 According to an Agilis Biotherapeutics media release, this pivotal study in anticipated to begin in 2017.
    • 02 Mar 2016 New trial record
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