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Myotonic Dystrophy Type 1

1
Pipeline Programs
4
Companies
4
Clinical Trials
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
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Avidity Biosciences
Avidity BiosciencesCA - San Diego
1 program
1
del-desiranPhase 32 trials
Active Trials
NCT07008469Enrolling By Invitation230Est. Oct 2030
NCT06411288Active Not Recruiting159Est. Sep 2026
Ionis Pharmaceuticals
1 program
IONIS-DMPKRxPHASE_1_21 trial
Active Trials
NCT02312011Completed48Est. Aug 2016
Insmed
InsmedBRIDGEWATER, NJ
1 program
rhIGF-I/rhIGFBP-3PHASE_21 trial
Active Trials
NCT00577577Completed69Est. Dec 2008

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
Avidity Biosciencesdel-desiran
Avidity Biosciencesdel-desiran
InsmedrhIGF-I/rhIGFBP-3
Ionis PharmaceuticalsIONIS-DMPKRx

Clinical Trials (4)

Total enrollment: 506 patients across 4 trials

Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Start: Jul 2025Est. completion: Oct 2030230 patients
Phase 3Enrolling By Invitation

Global Study of Del-desiran for the Treatment of DM1

Start: May 2024Est. completion: Sep 2026159 patients
Phase 3Active Not Recruiting
NCT00577577InsmedrhIGF-I/rhIGFBP-3

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Start: Dec 2007Est. completion: Dec 200869 patients
Phase 2Completed

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

Start: Dec 2014Est. completion: Aug 201648 patients
Phase 1/2Completed

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
4 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.