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A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

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Trial Profile

A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 15 Mar 2025

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At a glance

  • Drugs GC 101 (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors GeneCradle Therapeutics

Most Recent Events

  • 11 Sep 2023 Status changed from not yet recruiting to recruiting.
  • 15 Jun 2023 New trial record

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