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Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study

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Trial Profile

Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study

Status: Completed
Phase of Trial: Phase IV

Latest Information Update: 27 Feb 2024

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At a glance

  • Drugs Velaglucerase alfa (Primary)
  • Indications Gaucher's disease type I; Gaucher's disease type III
  • Focus Therapeutic Use
  • Acronyms PEDS
  • Sponsors Takeda

Most Recent Events

  • 22 Feb 2024 Status changed from recruiting to completed.
  • 05 Jan 2023 Planned End Date changed from 2 Jan 2023 to 2 Jan 2025.
  • 05 Jan 2023 Planned primary completion date changed from 2 Jan 2023 to 2 Jan 2025.

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