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
Latest Information Update: 25 Mar 2025
At a glance
- Drugs SAR 446268 (Primary)
- Indications Myotonic dystrophy
- Focus Adverse reactions
- Sponsors Sanofi
- 25 Mar 2025 New trial record