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A Phase 1/Phase 2 Open-label Single Arm Study With Dose Escalation (Part A), and Dose Expansion (Part B) Parts to Evaluate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants 10 to 50 Years Old With Non-congenital Myotonic Dystrophy Type 1

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Trial Profile

A Phase 1/Phase 2 Open-label Single Arm Study With Dose Escalation (Part A), and Dose Expansion (Part B) Parts to Evaluate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants 10 to 50 Years Old With Non-congenital Myotonic Dystrophy Type 1

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

Latest Information Update: 25 Mar 2025

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At a glance

  • Drugs SAR 446268 (Primary)
  • Indications Myotonic dystrophy
  • Focus Adverse reactions
  • Sponsors Sanofi

Most Recent Events

  • 25 Mar 2025 New trial record

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