Records containing: Myotonic Dystrophy Type 1 (DM1)
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CLINICAL_TRIALS study results posted · 2026-09-28A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
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CLINICAL_TRIALS study record update · 2026-09-01A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2026-04-21A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2026-04-01REACH DM - Remote Assessments and Genetic Determinants of Myotonic Dystrophy
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CLINICAL_TRIALS study record update · 2026-03-12A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
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CLINICAL_TRIALS study record update · 2026-03-05A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2026-02-27An Open-label Extension Study Evaluating the Long-term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type I
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CLINICAL_TRIALS study record update · 2026-02-19Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)
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CLINICAL_TRIALS study record update · 2026-02-18A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2026-01-22A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2025-05-18A Registered Observational Cohort Study of Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2025-04-27Personalized Exercise Training for People With Rare Neuromuscular Disorders: a Multi-center, Evaluator-blinded, Two Arm, Randomized Controlled Study to Assess the Effects on Physical Function From Personalized Strength and Balance Exercise in a Rehabilitation Setting.
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CLINICAL_TRIALS study record update · 2025-03-28A Remote Physical Activity Program Via the PACE Tool to Counter the Physical Impairments, Accentuated by the Pandemic, in the Population Suffering from Type 1 Myotonic Dystrophy
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CLINICAL_TRIALS study record update · 2024-05-14Technology Assisted Rehabilitation for Upper Limb Function in Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2022-08-24Nociceptive Pain in Neuromuscular Disorders - Low Interventional Pilot Study to Assess Musculoskeletal Pain in Neuromuscular Disorders (NMD)
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CLINICAL_TRIALS study record update · 2022-06-15Characters) Dyspnea Assessment in Adult Patients With Myotonic Dystrophy Type 1: a Monocentric Pilot Study
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CLINICAL_TRIALS study record update · 2018-07-18Brain Involvement in Myotonic Dystrophy Type I: From Functional Neuroimaging to the Impact on Quality of Life