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A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1

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Trial Profile

A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1

Status: Not yet recruiting
Phase of Trial: Phase I/II

Latest Information Update: 30 Jul 2025

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At a glance

  • Drugs GB 221 Gemma Biotherapeutics (Primary)
  • Indications Spinal muscular atrophy
  • Focus Adverse reactions; Pharmacodynamics; Therapeutic Use
  • Sponsors GEMMA Biotherapeutics

Most Recent Events

  • 30 Jul 2025 New trial record

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