Duchenne Muscular Dystrophy
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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 trial_result · 2025-09-15Clinical Trial Update: Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study [NA]
This completed randomized controlled trial evaluated the effects of functional inspiratory muscle training with tele-rehabilitation on respi…
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CLINICAL_TRIALS trial_result · 2025-09-11Clinical Trial Update: AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. [Phase 1, Phase 2]
Phase 1/2 clinical trial of an AAV9 U7snRNA gene therapy for Duchenne muscular dystrophy caused by exon 2 duplications has completed, with r…
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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 withdrawal · 2025-09-04Clinical Trial Update: A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74 [Phase 1]
This clinical trial update reports that Sarepta Therapeutics' gene transfer therapy Delandistrogene moxeparvovec (SRP-9001) for Duchenne Mus…
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CLINICAL_TRIALS trial_result · 2025-08-27Clinical Trial Update: IRM Cardiaque en Respiration Libre Pour Des Patients Atteints de Dystrophinopathie sévère [NA]
A completed clinical trial evaluating free-breathing cardiac MRI in patients with Duchenne muscular dystrophy or Becker muscular dystrophy.
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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 trial_result · 2025-08-15Clinical Trial Update: Repurposing Empagliflozin for DMD-associated Cardiomyopathy in Children 6-18 Years of Age [Phase 2]
Phase 2 clinical trial protocol repurposing empagliflozin for Duchenne muscular dystrophy–associated dilated cardiomyopathy in children aged…
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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 trial_result · 2025-08-14Clinical Trial Update: Assessment of Neurodevelopmental Needs in Duchenne Muscular Dystrophy
A completed clinical trial conducted by Virginia Commonwealth University assessed neurodevelopmental needs in Duchenne muscular dystrophy.
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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 trial_result · 2025-07-14Clinical Trial Update: Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping [Phase 1, Phase 2]
Completed Phase 1/2 clinical trial of AOC 1044 in healthy adult volunteers and Duchenne muscular dystrophy patients with exon 44 skipping am…
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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 withdrawal · 2025-06-24Clinical Trial Update: A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment [Phase 2]
A Phase 2 study of PGN-EDO51 or placebo for Duchenne muscular dystrophy amenable to exon 51 skipping has been withdrawn.