Myotonic Dystrophy Type
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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 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 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 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 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 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…
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CLINICAL_TRIALS research · 2025-11-10Clinical Trial Update: Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy [Phase 1, Phase 2]
Ongoing Phase 1/2 clinical trial evaluating ARO-DM1 in subjects with Myotonic Dystrophy Type 1, currently recruiting.
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CLINICAL_TRIALS research · 2025-10-15Clinical Trial Update: Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry
A registry study update listing RECRUITING status for Myotonic Dystrophy type 1, Myotonic Dystrophy type 2, and Facioscapulohumeral muscular…
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CLINICAL_TRIALS research · 2025-09-16Clinical Trial Update: A Multicenter Phenotype-Genotype Analysis of DM1 Patients in China
A multicenter study from Huashan Hospital in China is conducting a phenotype-genotype analysis of patients with myotonic dystrophy type 1. T…
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CLINICAL_TRIALS research · 2025-08-22Clinical Trial Update: Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology [NA]
This clinical trial update describes the assessment of gait analysis parameters and upper-limb evaluation in adults with various neurologica…
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CLINICAL_TRIALS research · 2025-07-30Clinical Trial Update: Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension
This clinical trial extension outlines plans to assess endpoints and trial readiness for a pediatric myotonic dystrophy study. It targets co…
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CLINICAL_TRIALS research · 2025-07-18Clinical Trial Update: The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period. [NA]
This clinical trial update outlines a study evaluating whether assistive gait devices, following a training period, can reduce fall risk in …
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CLINICAL_TRIALS research · 2025-06-11Clinical Trial Update: DMCRN-02-001: Assessing Pediatric Endpoints in DM1
A clinical trial (DMCRN-02-001) assessing pediatric endpoints in congenital myotonic dystrophy (DM1) is currently recruiting.
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CLINICAL_TRIALS research · 2025-06-06Clinical Trial Update: Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
This clinical trial update describes END-DM1, a Virginia Commonwealth University study to establish biomarkers and clinical endpoints for my…
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CLINICAL_TRIALS research · 2025-05-28Clinical Trial Update: Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy [Phase 2, Phase 3]
This clinical trial update notes a Phase 2/3 study evaluating tideglusib for congenital or childhood-onset myotonic dystrophy that is curren…