Duchenne Muscular Dystrophy
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CLINICAL_TRIALS research · 2025-11-25Clinical Trial Update: AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This clinical trial update describes the AFFINITY BEYOND study, an anti-AAV8 antibody assessment in males with Duchenne muscular dystrophy, …
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
A clinical trial at Massachusetts General Hospital is recruiting to study extracellular RNA biomarkers in Duchenne Muscular Dystrophy. The s…
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CLINICAL_TRIALS research · 2025-11-24Clinical Trial Update: Biomarker Development for Muscular Dystrophies
Massachusetts General Hospital sponsors a clinical trial update focused on developing biomarkers for muscular dystrophy. The study is curren…
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy [Phase 2]
Phase 2 study of EDG-5506 in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy. The trial is cu…
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CLINICAL_TRIALS research · 2025-11-06Clinical Trial Update: Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim
A longitudinal clinical trial update from Vanderbilt University Medical Center describes using wearable technology to monitor hyperglycemia …
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CLINICAL_TRIALS research · 2025-10-15Clinical Trial Update: Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
This clinical trial investigates MRI techniques and biomarkers for muscular dystrophy. It is currently recruiting and sponsored by the Unive…
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: Safety and Dystrophin Expression of SPOT-mRNA03 in Duchenne Muscular Dystrophy (DMD) Patients [EARLY_Phase 1]
This early-phase 1 trial is evaluating the safety and dystrophin expression of SPOT-mRNA03 in patients with Duchenne muscular dystrophy and …
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CLINICAL_TRIALS research · 2025-09-19Clinical Trial Update: Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy [Phase 2]
This update concerns a long-term extension study of DS-5141b in Duchenne muscular dystrophy; the trial is currently active but not recruitin…
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CLINICAL_TRIALS research · 2025-09-11Clinical Trial Update: A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD） [Phase 3]
This clinical-trial update covers the Phase 3 REACH-DMD study evaluating TAS-205 in Duchenne muscular dystrophy, noting that the trial is cu…
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CLINICAL_TRIALS research · 2025-09-10Clinical Trial Update: A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
This update describes a long-term observational study evaluating eteplirsen, golodirsen, and casimersen in routine clinical practice for Duc…
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CLINICAL_TRIALS research · 2025-08-19Clinical Trial Update: Fear of Falling in Muscular Dystrophy [NA]
A Verona, Italy–based clinical trial is currently recruiting to investigate fear of falling in individuals with Duchenne muscular dystrophy.
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CLINICAL_TRIALS research · 2025-08-15Clinical Trial Update: Decoding Central Defects in Dystrophinopathies From Diagnostic to Remediation
Clinical trial update describing a study on central defects in dystrophinopathies, from diagnostic assessment to remediation strategies. The…
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CLINICAL_TRIALS research · 2025-08-13Clinical Trial Update: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping [Phase 1, Phase 2]
A Phase 1/2 clinical trial update evaluating the safety, tolerability, pharmacodynamics, efficacy, and pharmacokinetics of DYNE-251 in Duche…
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CLINICAL_TRIALS research · 2025-08-13Clinical Trial Update: Once Weekly Infant Corticosteroid Trial for DMD [Phase 4]
Phase 4 clinical trial evaluating once-weekly corticosteroid therapy in infants with Duchenne Muscular Dystrophy. The study is currently rec…
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CLINICAL_TRIALS research · 2025-08-11Clinical Trial Update: Wearable Technology to Evaluate Hyperglycemia and HRV in DMD
This clinical trial is investigating wearable technology to measure hyperglycemia and heart rate variability in patients with Duchenne Muscu…
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CLINICAL_TRIALS research · 2025-08-03Clinical Trial Update: Transition to Adulthood in People With Muscular Dystrophy [NA]
Clinical trial update on transition to adulthood in people with Duchenne muscular dystrophy and Becker muscular dystrophy; the study is curr…
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CLINICAL_TRIALS research · 2025-07-31Clinical Trial Update: Parent and Infant Inter(X)Action Intervention (PIXI) [NA]
Clinical trial update for the PIXI Inter(X)Action Intervention (NCT03836300), currently enrolling by invitation across Fragile X Syndrome, A…
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CLINICAL_TRIALS research · 2025-07-29Clinical Trial Update: A Study to Evaluate the Safety and Tolerability of GEN6050X in Duchenne Muscular Dystrophy. [EARLY_Phase 1]
An early-phase study evaluating the safety and tolerability of GEN6050X in Duchenne muscular dystrophy, with the trial currently active but …
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CLINICAL_TRIALS research · 2025-07-20Clinical Trial Update: Extension Study of NS-089/NCNP-02 in DMD [Phase 2]
The extension study of NS-089/NCNP-02 for Duchenne muscular dystrophy is described as a Phase 2 trial. The study is currently active but not…
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CLINICAL_TRIALS research · 2025-07-10Clinical Trial Update: Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old [Phase 2]
Phase 2 clinical trial assessing pharmacokinetics and safety of givinostat in children with Duchenne muscular dystrophy aged 2 to under 6 ye…
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CLINICAL_TRIALS research · 2025-07-09Clinical Trial Update: Vasodilator and Exercise Study for DMD (VASO-REx) [Phase 2]
A Phase 2 trial named VASO-REx is evaluating a vasodilator in combination with exercise for Duchenne muscular dystrophy and is currently rec…
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CLINICAL_TRIALS research · 2025-06-26Clinical Trial Update: The Effect of Virtual Reality Applications on Upper Extremity Functions in Patients With Duchenne Muscular Dystrophy [NA]
This clinical trial update describes a study evaluating virtual reality applications to improve upper-extremity function in Duchenne muscula…
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CLINICAL_TRIALS research · 2025-06-13Clinical Trial Update: ML-Based Multi-Sensor Fall Risk Screening in DMD
Clinical trial update on a machine-learning based multi-sensor fall risk screening tool for Duchenne muscular dystrophy; the study is curren…
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CLINICAL_TRIALS research · 2025-06-06Clinical Trial Update: Follow-up Study on Female Carriers With DMD Gene Variants
Clinical trial update describing a follow-up study enrolling female carriers of DMD gene variants to assess clinical characteristics related…
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CLINICAL_TRIALS research · 2025-05-20Clinical Trial Update: Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study [NA]
A clinical trial at Centre Hospitalier Universitaire de Liege assesses muscular pathology in patients and controls under the ActiLiège Next …