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Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease

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Trial Profile

Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 07 Nov 2023

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At a glance

  • Drugs GC-301 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions

Most Recent Events

  • 11 Oct 2022 New trial record

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