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A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease

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Trial Profile

A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 08 Jun 2023

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At a glance

  • Drugs GC-301 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors GeneCradle Therapeutics

    • Most Recent Events

    • 04 Jun 2023 Status changed from not yet recruiting to recruiting.
    • 03 Apr 2023 New trial record

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