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Myotonic Dystrophy Type 1 (DM1)

0
Pipeline Programs
2
Companies
4
Clinical Trials
3 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Vertex Pharmaceuticals
2 programs
VX-670PHASE_1_21 trial
VX-670PHASE_21 trial
Active Trials
NCT06185764Recruiting44Est. Dec 2026
NCT06926621Enrolling By Invitation44Est. Jan 2029
Dyne Therapeutics
1 program
DYNE-101PHASE_1_22 trials
Active Trials
NCT07486934Recruiting150Est. Jan 2029
NCT05481879Recruiting116Est. Jul 2029

Trial Timeline

Clinical trial activity over time

2022
2023
2024
2025
2026
2027
2028
2029
Dyne TherapeuticsDYNE-101
Vertex PharmaceuticalsVX-670
Vertex PharmaceuticalsVX-670
Dyne TherapeuticsDYNE-101

Clinical Trials (4)

Total enrollment: 354 patients across 4 trials

NCT07486934Dyne TherapeuticsDYNE-101

Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1

Start: Apr 2026Est. completion: Jan 2029150 patients
Phase 3Recruiting
NCT06926621Vertex PharmaceuticalsVX-670

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

Start: Apr 2025Est. completion: Jan 202944 patients
Phase 2Enrolling By Invitation
NCT06185764Vertex PharmaceuticalsVX-670

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Start: Feb 2024Est. completion: Dec 202644 patients
Phase 1/2Recruiting
NCT05481879Dyne TherapeuticsDYNE-101

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Start: Sep 2022Est. completion: Jul 2029116 patients
Phase 1/2Recruiting

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 actively recruiting trials targeting 354 patients
2 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.