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Open-label extension study of VX-670 in adults with myotonic dystrophy type 1
2024-517983-47-00Phase 2Authorised
- Sponsor
- Vertex Pharmaceuticals
- Start
- Nov 2025
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 10
01
Overview
Phase 2, open-label, single-arm trial of VX-670 in myotonic dystrophy type 1 (DM1), sponsored by Vertex Pharmaceuticals. Primary endpoint: Safety and tolerability of VX-670. Planned start Nov 2025; status: authorised.
02
Drugs
VX-670
03
Conditions
myotonic dystrophy type 1 (DM1)
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