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Long-term extension study of VX-670 in myotonic dystrophy type 1
NL-OMON59875Phase 2Recruiting
- Sponsor
- Vertex Pharmaceuticals
- Start
- Apr 2026
- 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
- 1
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. Started Apr 2026; status: recruiting.
02
Drugs
VX-670
03
Conditions
myotonic dystrophy type 1 (DM1)
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