Myotonic Dystrophy 1
2
Pipeline Programs
4
Companies
3
Clinical Trials
1 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
1
1
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Harmony BiosciencesPitolisant Oral Tablet
Arrowhead PharmaceuticalsARO-DM1 Intravenous
Life & Brainmedical history
Clinical Trials (3)
Total enrollment: 108 patients across 3 trials
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
Start: Jun 2021Est. completion: Oct 202430 patients
Phase 2Completed
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Start: Mar 2024Est. completion: Dec 202678 patients
Phase 1/2Recruiting
Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study
Start: May 2007Est. completion: Aug 2015
N/ACompleted
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
1 actively recruiting trials targeting 108 patients
4 companies competing in this space