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VX-670 phase 1/2 study in adults with myotonic dystrophy type 1
NL-OMON60156Phase 2Recruiting
- Sponsor
- Vertex Pharmaceuticals
- Start
- Dec 2024
- 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 trial of VX-670 in myotonic dystrophy type 1 (DM1) (2 arms), sponsored by Vertex Pharmaceuticals. Primary endpoint: Safety and tolerability. Started Dec 2024; status: recruiting.
02
Drugs
VX-670
03
Conditions
myotonic dystrophy type 1 (DM1)
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