Records containing: Muscular Dystrophy, Duchenne
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CLINICAL_TRIALS study record update · 2026-06-05A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2026-04-27Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping
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CLINICAL_TRIALS study record update · 2026-04-23Evaluation of the Diagnostic Performance of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
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CLINICAL_TRIALS study record update · 2026-04-08Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping
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CLINICAL_TRIALS study record update · 2026-04-06A Real-World, Multicenter Cohort Study on the Natural History of Duchenne and Becker Muscular Dystrophy in Children From Western China
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CLINICAL_TRIALS study record update · 2026-04-06An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
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CLINICAL_TRIALS study record update · 2026-03-10A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
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CLINICAL_TRIALS study record update · 2026-02-27A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
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CLINICAL_TRIALS study record update · 2026-02-24Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care
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CLINICAL_TRIALS study record update · 2025-12-17A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2025-12-03The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder
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CLINICAL_TRIALS study record update · 2025-10-21A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY
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CLINICAL_TRIALS study record update · 2025-03-05To Train -or Not to Train? the Role of Physical Exercise As Part of Management in Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2025-02-24A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2025-02-18Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
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CLINICAL_TRIALS study record update · 2024-09-19An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
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CLINICAL_TRIALS study record update · 2024-03-29Characterization of the Phenotypic Diversity in DupEx2 Duchenne Muscular Dystrophy and Identification of Predictive/Prognostic Markers
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CLINICAL_TRIALS study record update · 2023-10-23A Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid-naïve Boys With DMD
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CLINICAL_TRIALS study record update · 2023-09-15A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
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CLINICAL_TRIALS study record update · 2023-05-15Study of Genotype and Phenotype Characterization and Biomarkers Profile in Duchenne Muscular Dystrophy With Small Mutations
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CLINICAL_TRIALS study record update · 2022-09-23A Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2022-09-09Effects of Parental Influence on Physical Activity Level And Participation in Children With Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2022-07-06A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment
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CLINICAL_TRIALS study record update · 2022-06-21A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy
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CLINICAL_TRIALS study record update · 2022-05-10Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for Duchenne Muscular Dystrophy (DMD)