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Phase 1/2 Study of VX-670 in Adults with Myotonic Dystrophy Type 1
2023-506028-10-00Phase 2Authorised
- Sponsor
- Vertex Pharmaceuticals
- Start
- Mar 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
- 10
01
Overview
Phase 2, randomised, double-blind trial of VX-670 in myotonic dystrophy type 1 (DM1) (4 arms), sponsored by Vertex Pharmaceuticals. Primary endpoint: Safety and tolerability of VX-670. Planned start Mar 2024; status: authorised.
02
Drugs
VX-670
03
Conditions
myotonic dystrophy type 1 (DM1)
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