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 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 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 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 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 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 trial_result · 2025-11-24Clinical Trial Update: Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy
A Massachusetts General Hospital clinical trial is investigating cerebrospinal fluid biomarkers in myotonic dystrophy type 1. The study is c…
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CLINICAL_TRIALS trial_result · 2025-10-08Clinical Trial Update: Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients [Phase 2]
This clinical trials update reports the extension of the AOC 1001-CS1 (MARINA) study in adults with myotonic dystrophy type 1 (DM1). The Pha…
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CLINICAL_TRIALS trial_result · 2025-09-12Clinical Trial Update: Venous Thromboembolism in Myotonic Dystrophy Type 1 [NA]
The clinical trial NCT03424460 evaluating venous thromboembolism risk in patients with Myotonic Dystrophy Type 1 has completed. The sponsor …
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CLINICAL_TRIALS trial_result · 2025-09-11Clinical Trial Update: Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy [Phase 2]
A Phase 2 study of Tideglusib in adolescent and adult patients with myotonic dystrophy type 1 has completed and results have been posted.
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CLINICAL_TRIALS trial_result · 2025-08-19Clinical Trial Update: Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)
This clinical trial update describes the profile of swallowing difficulties (dysphagia) in individuals with Myotonic Dystrophy Type 1 and no…
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CLINICAL_TRIALS trial_result · 2025-04-27Clinical Trial Update: Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
A completed clinical study evaluating symptoms and outcome measures for upper-limb function in individuals with myotonic dystrophy type 1.
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CLINICAL_TRIALS trial_result · 2025-04-25Clinical Trial Update: Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 [Phase 2]
A Phase 2 trial evaluated the safety and efficacy of pitolisant for excessive daytime sleepiness and other non-muscular symptoms in adults w…
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CLINICAL_TRIALS trial_result · 2024-07-05Clinical Trial Update: Trial Readiness and Endpoint Assessment in Congenital and Childhood Myotonic Dystrophy
Completed clinical trial update focusing on trial readiness and endpoint assessment in congenital and childhood myotonic dystrophy.
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CLINICAL_TRIALS trial_result · 2024-05-14Clinical Trial Update: Technology Assisted Rehabilitation for Upper Limb Function in Myotonic Dystrophy Type 1 [NA]
A completed clinical trial evaluating technology-assisted rehabilitation for improving upper limb function in people with Myotonic Dystrophy…
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CLINICAL_TRIALS trial_result · 2023-10-19Clinical Trial Update: Home-based Training and Supplementation in DM1 Patients [Phase 3]
A Phase 3 clinical trial evaluating home-based training and supplementation in patients with Myotonic Dystrophy Type 1 is ongoing (status un…
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CLINICAL_TRIALS trial_result · 2022-12-06Clinical Trial Update: A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 [Phase 1, Phase 2]
A completed Phase 1/2 study evaluating safety and tolerability of multiple doses of ISIS-DMPKRx in adults with myotonic dystrophy type 1.
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CLINICAL_TRIALS trial_result · 2022-01-06Clinical Trial Update: 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 [Phase 2]
Phase 2 trial assessing safety and efficacy of rhIGF-I/rhIGFBP-3 in Myotonic Dystrophy Type 1 has completed with results posted.
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CLINICAL_TRIALS trial_result · 2021-06-23Clinical Trial Update: Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1 [Phase 1]
Phase 1 study assessing safety, tolerability, and pharmacokinetics of ERX-963 in adults with myotonic dystrophy type 1 has been completed wi…
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CLINICAL_TRIALS trial_result · 2018-07-18Clinical Trial Update: Brain Involvement in Myotonic Dystrophy Type I: From Functional Neuroimaging to the Impact on Quality of Life
This study investigated brain involvement in Myotonic Dystrophy Type 1 using functional neuroimaging to assess its impact on quality of life…
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CLINICAL_TRIALS trial_result · 2018-07-11Clinical Trial Update: Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy [NA]
This clinical trial update reports on ventilatory response after non-invasive ventilation in patients with Myotonic Dystrophy Type 1 (Steine…
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CLINICAL_TRIALS trial_result · 2018-03-29Clinical Trial Update: Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1 [Phase 2]
This document summarizes a Phase 2 clinical efficacy trial of mexiletine for myotonic dystrophy type 1. Results were posted in 2018.
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CLINICAL_TRIALS trial_result · 2017-05-09Clinical Trial Update: Venous Thromboembolism in DM1
This clinical trial update reports the completed status of a study evaluating venous thromboembolism in myotonic dystrophy type 1.