Records containing: Myotonic Dystrophy 1
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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 · 2026-04-23A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2026-03-30An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
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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 · 2026-03-02A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)
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CLINICAL_TRIALS study record update · 2026-02-13A Phase 1 Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending Doses of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM-DM1)
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CLINICAL_TRIALS study record update · 2026-02-10A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1
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CLINICAL_TRIALS study record update · 2026-02-04Creación de un Nodo Integral Para la Distrofia Miotónica Tipo 1 en España: Registro clínico, Mapas genómicos, epigenómicos y proteómicos (DM1-Hub)
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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 · 2025-12-09Calcium Channel Blocker in Myotonic Dystrophy Type 1 (CAP DM1)
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CLINICAL_TRIALS study record update · 2025-11-24Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease). A Phase III, Prospective, Multicentre, Randomized, Double-blind Controlled Study
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CLINICAL_TRIALS study record update · 2025-11-10A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years
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CLINICAL_TRIALS study record update · 2025-10-08A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
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CLINICAL_TRIALS study record update · 2025-09-16A Multicenter Phenotype-Genotype Analysis of Type 1 Myotonic Dystrophy 1 Patients in China
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CLINICAL_TRIALS study record update · 2025-09-12Venous Thromboembolism in Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2025-09-11A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy
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CLINICAL_TRIALS study record update · 2025-08-22Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
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CLINICAL_TRIALS study record update · 2025-08-19A Multidimensional Profile of Dysphagia in People Living With Myotonic Dystrophy Type 1 (plwDM1)
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CLINICAL_TRIALS study record update · 2025-07-18The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
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CLINICAL_TRIALS study record update · 2025-06-06Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
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CLINICAL_TRIALS study record update · 2025-05-23Links Between Diabetes and Cognitive Impairment in Myotonic Dystrophy Type 1 : a Non-conventional MRI Study
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CLINICAL_TRIALS study record update · 2025-05-20Gait Analysis Parameter, Stair Climbing and Upper Limb Evaluation in Patients With Muscular Pathology and in Control Subjects: The ActiLiège Next Study
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CLINICAL_TRIALS study record update · 2025-04-27Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1
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CLINICAL_TRIALS study record update · 2025-04-25A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension
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CLINICAL_TRIALS study record update · 2024-11-21Myotonic Dystrophy Family Registry