Myotonic Dystrophy Type
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CLINICAL_TRIALS trial_result · 2026-04-23Clinical Trial Update: Global Open-Label Extension Study of Del-desiran for the Treatment of DM1 [Phase 3]
This document reports on a global open-label extension study (Phase 3) evaluating Del-desiran for myotonic dystrophy type 1, with enrollment…
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1 [Phase 3]
Phase 3 clinical trial update indicates Dyne Therapeutics is evaluating Zeleciment Basivarsen (DYNE-101) for efficacy, safety, and tolerabil…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: Investigating Exercise in Myotonic Dystrophy Type 2 (DM2) [NA]
An ongoing Massachusetts General Hospital clinical trial is examining the effects of exercise in myotonic dystrophy type 2 and is currently …
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CLINICAL_TRIALS research · 2026-04-01Clinical Trial Update: Remote Assessments and Genetic Determinants of Myotonic Dystrophy
A University of Rochester-sponsored clinical trial on remote assessments and genetic determinants in Myotonic Dystrophy Type 1 is currently …
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) [Phase 2]
An open-label extension study (FREEDOM-OLE) of the investigational drug PGN-EDODM1 in people with Myotonic Dystrophy Type 1, a Phase 2 trial…
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CLINICAL_TRIALS research · 2026-03-25Clinical Trial Update: Fall Frequency and Factors Affecting Dynamic Balance in Patients With Myotonic Dystrophy Type 1
Status update for a clinical trial evaluating fall frequency and dynamic balance in patients with Myotonic Dystrophy Type 1, sponsored by An…
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CLINICAL_TRIALS trial_result · 2026-03-12Clinical Trial Update: Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients [Phase 1, Phase 2]
The Phase 1/2 clinical trial of AOC 1001 in adults with Myotonic Dystrophy Type 1 has completed.
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CLINICAL_TRIALS research · 2026-03-05Clinical Trial Update: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 [Phase 1, Phase 2]
This entry describes an ongoing Phase 1/Phase 2 clinical trial of DYNE-101 in Myotonic Dystrophy Type 1 assessing safety, tolerability, phar…
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CLINICAL_TRIALS research · 2026-03-02Clinical Trial Update: A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 [Phase 2]
PepGen’s Phase 2 clinical study of PGN-EDODM1 for myotonic dystrophy type 1 is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-02-27Clinical Trial Update: A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I [Phase 2]
Phase 2 study evaluating long-term safety and efficacy of VX-670 in patients with Myotonic Dystrophy Type 1; the trial is currently enrollin…
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CLINICAL_TRIALS research · 2026-02-25Clinical Trial Update: Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies [NA]
A clinical trial is recruiting to evaluate the role of microRNA-1 (miR-1) in disease pathogenesis and as a biomarker for muscular dystrophie…
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CLINICAL_TRIALS research · 2026-02-19Clinical Trial Update: Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1) [NA]
This clinical trial update describes a study investigating a music intervention as a potential non-pharmacologic approach for brain-heart di…
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CLINICAL_TRIALS research · 2026-02-18Clinical Trial Update: A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating VX-670 in adults with Myotonic Dystrophy Type 1 is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-02-13Clinical Trial Update: Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1 [Phase 1]
Phase 1 study evaluating safety, tolerability, pharmacokinetics, and pharmacodynamics of PGN-EDODM1 in participants with Myotonic Dystrophy …
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CLINICAL_TRIALS trial_result · 2026-02-10Clinical Trial Update: Study of ATX-01 in Participants With DM1 [Phase 1, Phase 2]
ARTHEx Biotech is conducting a Phase 1/2 trial of ATX-01 in participants with Myotonic Dystrophy Type 1, and recruitment is currently ongoin…
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CLINICAL_TRIALS research · 2026-02-06Clinical Trial Update: The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 [Phase 3]
This clinical trial update describes a Phase 3 study evaluating once-daily mexiletine prolonged-release for patients with myotonic dystrophy…
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: The Spanish National Registry for Myotonic Dystrophy Type 1
The Spanish National Registry for Myotonic Dystrophy Type 1 (NCT07385443) is sponsored by Fundació Institut Germans Trias i Pujol and is cur…
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CLINICAL_TRIALS research · 2026-02-02Clinical Trial Update: Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
A clinical trial assessing brain structure and clinical endpoints in myotonic dystrophy type 2 is currently recruiting.
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CLINICAL_TRIALS other · 2026-02-02Clinical Trial Update: A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1 [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/2 study evaluating the safety, tolerability, and efficacy of SAR446268, an adeno-associated v…
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CLINICAL_TRIALS trial_result · 2026-01-26Clinical Trial Update: An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study. [Phase 3]
An open-label extension study is evaluating the long-term safety and efficacy of once-daily Mexiletine prolonged-release in patients with my…
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CLINICAL_TRIALS trial_result · 2026-01-23Clinical Trial Update: Longitudinal Evaluation of Neuromuscular Involvement in Type 1 Myotonic Dystrophy
This clinical trial update notes the completion of a longitudinal evaluation of neuromuscular involvement in myotonic dystrophy type 1 (Stei…
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CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: Global Study of Del-desiran for the Treatment of DM1 [Phase 3]
Phase 3 global trial of Del-desiran for Myotonic Dystrophy Type 1 (DM1) is currently not recruiting.
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CLINICAL_TRIALS research · 2025-12-09Clinical Trial Update: Calcium Channel Blocker in Myotonic Dystrophy Type 1 [Phase 1]
A Phase 1 clinical trial evaluating a calcium channel blocker for myotonic dystrophy type 1 is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2025-12-05Clinical Trial Update: Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years
This completed clinical trial study identifies factors linked to hypoventilation in people with Myotonic Dystrophy Type 1 over a five-year f…
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) [Phase 3]
Phase 3 clinical trial evaluating the efficacy and safety of metformin in myotonic dystrophy type 1 (Steinert's disease) is currently recrui…