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A phase 2 open label study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease

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Trial Profile

A phase 2 open label study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 10 Oct 2024

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At a glance

  • Drugs Vestronidase alfa (Primary)
  • Indications Mucopolysaccharidosis VII
  • Focus Therapeutic Use

    • Most Recent Events

    • 21 Dec 2020 Status changed from active, no longer recruiting to completed.
    • 01 Jun 2020 Status changed from recruiting to active, no longer recruiting.
    • 12 Sep 2019 Planned End Date changed from 31 Aug 2019 to 31 Dec 2020.

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