Duchenne Muscular Dystrophy
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CLINICAL_TRIALS research · 2025-05-14Clinical Trial Update: Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping [Phase 2]
A Phase 2 open-label study evaluating AOC-1044 for Duchenne muscular dystrophy with mutations amenable to exon 44 skipping is currently acti…
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CLINICAL_TRIALS research · 2025-05-11Clinical Trial Update: Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy [Phase 3]
A Phase 3 trial evaluating the efficacy, safety and tolerability of givinostat in non-ambulant Duchenne muscular dystrophy patients; current…
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CLINICAL_TRIALS research · 2025-04-30Clinical Trial Update: DMD Voice: Qualitative Interviews With Patients and Caregivers
A ClinicalTrials.gov entry update describes a recruiting qualitative study called 'DMD Voice' that interviews patients with Duchenne Muscula…
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CLINICAL_TRIALS research · 2025-04-04Clinical Trial Update: Early Check: Expanded Screening in Newborns
An update on an ongoing clinical trial (NCT03655223) evaluating expanded newborn screening under the Early Check program, with enrollment by…
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CLINICAL_TRIALS research · 2025-04-02Clinical Trial Update: Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy [EARLY_Phase 1]
Early-phase clinical trial update on LE051 evaluating safety, tolerability, and potential efficacy in Duchenne Muscular Dystrophy; the study…
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CLINICAL_TRIALS research · 2025-04-01Clinical Trial Update: Effectiveness of 5-week Digital Respiratory Practice in Children With Duchenne and Becker Muscular Dystrophies. [NA]
A clinical trial update evaluating the effectiveness of a 5-week digital respiratory practice program in children with Duchenne muscular dys…
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CLINICAL_TRIALS research · 2025-03-28Clinical Trial Update: Psychoeducation Program for Parents of Children with Duchenne Muscular Dystrophy [NA]
This clinical trial update covers a psychoeducation program for parents of children with Duchenne muscular dystrophy, with participant recru…
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CLINICAL_TRIALS research · 2025-03-25Clinical Trial Update: Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy [EARLY_Phase 1]
An early-phase clinical trial is evaluating the safety and potential efficacy of BBM-D101 for Duchenne muscular dystrophy. The study is curr…
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CLINICAL_TRIALS research · 2025-03-21Clinical Trial Update: NSAA NON-Interventional Study Protocol
This document provides an update on a non-interventional study protocol using NSAA for Duchenne muscular dystrophy and notes that the study …
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CLINICAL_TRIALS research · 2025-03-13Clinical Trial Update: Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders
A clinical trial update notes a NOT_YET_RECRUITING study examining social cognition in Duchenne muscular dystrophy, Becker muscular dystroph…
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CLINICAL_TRIALS research · 2025-03-06Clinical Trial Update: Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France
This ClinicalTrials.gov study from Assistance Publique - Hôpitaux de Paris in France aims to estimate non-reimbursable costs for patients wi…
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CLINICAL_TRIALS research · 2025-03-05Clinical Trial Update: Physical Activity Level of Norwegian Boys with Duchenne Muscular Dystrophy
This clinical trial update reports on the physical activity level of Norwegian boys with Duchenne Muscular Dystrophy; the study status is co…
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CLINICAL_TRIALS research · 2025-02-27Clinical Trial Update: Tadalafil as Adjuvant Therapy for DMD [Phase 2, Phase 3]
Clinical trial update indicating tadalafil is being studied as adjuvant therapy for Duchenne Muscular Dystrophy in Phase 2/3 trials, with ac…
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CLINICAL_TRIALS research · 2025-02-18Clinical Trial Update: Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases [NA]
A recruiting clinical trial using transcriptomic analysis to reduce misdiagnosis in patients with rare genetic muscle diseases, including Du…
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CLINICAL_TRIALS research · 2025-02-10Clinical Trial Update: The Duchenne Registry
The Duchenne Registry trial is currently recruiting, sponsored by The Duchenne Registry, and targets Duchenne muscular dystrophy and Becker …
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CLINICAL_TRIALS research · 2025-01-30Clinical Trial Update: Hydrotherapy in Duchenne Muscular Dystrophy (DMD) [NA]
This clinical trial update describes hydrotherapy for Duchenne Muscular Dystrophy, with the study currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-01-03Clinical Trial Update: Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
A pediatric neuromuscular disease trial is recruiting to evaluate respiratory function, thoracoabdominal movements, and exercise capacity.
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CLINICAL_TRIALS research · 2025-01-01Clinical Trial Update: Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among Patients with Duchenne Muscular Dystrophy.
A clinical trial is examining the relationships among scoliosis, pain, quality of life, and trunk muscle compensation in Duchenne muscular d…
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CLINICAL_TRIALS research · 2024-12-27Clinical Trial Update: Analysis of Motor Performance in Children with Duchene's Muscular Dystrophy
A clinical trial update from Cairo University describes planned analysis of motor performance in children with Duchenne muscular dystrophy, …
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CLINICAL_TRIALS research · 2024-12-16Clinical Trial Update: Pediatric Radio Frequency Coils Generic
Clinical trial update indicating an ongoing pediatric radio frequency coils generic study with status ACTIVE_NOT_RECRUITING.
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CLINICAL_TRIALS research · 2024-12-13Clinical Trial Update: Evaluation of Brain Dysfunction in Patients with Duchene Muscular Dystrophy
Clinical trial update evaluating brain dysfunction in patients with Duchenne Muscular Dystrophy; the study is currently not yet recruiting.
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CLINICAL_TRIALS research · 2024-11-14Clinical Trial Update: FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a longitudinal epidemiology study by xCures to follow outcomes across multiple rare diseases, and the study is currently recruitin…
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CLINICAL_TRIALS research · 2024-10-16Clinical Trial Update: Effects of Lumbopelvic Stabilization-based Physiotherapy and Rehabilitation Training in Duchenne Muscular Dystrophy [NA]
This clinical trial examines a lumbopelvic stabilization-based physiotherapy and rehabilitation program for Duchenne muscular dystrophy, wit…
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CLINICAL_TRIALS research · 2024-10-04Clinical Trial Update: phenotypeS in Non Ambulant Duchenne Muscular Dystrophy
The phenotypeS study in non-ambulant Duchenne muscular dystrophy is currently recruiting, according to the trial update.
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CLINICAL_TRIALS research · 2024-09-23Clinical Trial Update: Wearable Technology to Assess Gait Function in SMA and DMD
A completed clinical trial evaluating wearable technology to assess gait function in spinal muscular atrophy type 3 and Duchenne muscular dy…