Duchenne Muscular Dystrophy
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CLINICAL_TRIALS research · 2024-09-03Clinical Trial Update: Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies
A clinical trial update from University College London aims to define outcome measures for behavioral and emotional problems in dystrophinop…
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CLINICAL_TRIALS research · 2024-08-30Clinical Trial Update: Development of a Registry to Assess Natural History in Duchenne Muscular Dystrophy
This clinical trial update describes the development of a registry to characterize the natural history of Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS research · 2024-08-30Clinical Trial Update: Expanded Access Study of UC-MSC in DMD Patients
Expanded Access study update for UC-MSC therapy in Duchenne Muscular Dystrophy, with the study status listed as AVAILABLE.
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CLINICAL_TRIALS research · 2024-08-27Clinical Trial Update: Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
This clinical trial update characterizes skeletal and cardiac impairment in carriers of Duchenne muscular dystrophy and Becker muscular dyst…
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CLINICAL_TRIALS research · 2024-08-06Clinical Trial Update: Applying the Pathways and Resources for Engagement and Participation Protocol Among People With Muscles Dystrophies [NA]
Clinical trial update describing a protocol to apply engagement pathways and resources for participation among people with Duchenne Muscular…
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CLINICAL_TRIALS research · 2024-07-16Clinical Trial Update: Effect of Captopril on GLS in Duchenne Myodystrophy [EARLY_Phase 1]
An early-phase (Phase 1) clinical trial is evaluating the effect of captopril on global longitudinal strain in Duchenne muscular dystrophy, …
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CLINICAL_TRIALS research · 2019-12-05Clinical Trial Update: Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy [Phase 1, Phase 2]
Clinical trial update for antisense oligonucleotides in Duchenne Muscular Dystrophy (Phase 1/2) with completed status and results posted.
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CLINICAL_TRIALS research · 2018-05-15Clinical Trial Update: Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies [NA]
An update on a clinical trial evaluating muscle microRNAs as biomarkers in dystrophinopathies, including Duchenne and Becker muscular dystro…
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CLINICAL_TRIALS research · 2018-05-11Clinical Trial Update: Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy
A completed clinical trial investigating factors affecting hand functions in nonambulatory Duchenne muscular dystrophy patients.
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CLINICAL_TRIALS research · 2018-04-27Clinical Trial Update: Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dystrophy
Clinical trial update examining how fear of falling affects physical performance and quality of life in children with Duchenne Muscular Dyst…
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CLINICAL_TRIALS research · 2018-04-05Clinical Trial Update: The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
A completed clinical trial study examining the experiences of parents, clinician researchers, and the industry sponsor in the Ataluren (PTC1…
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CLINICAL_TRIALS research · 2018-03-16Clinical Trial Update: Biomechanical and Morphological Changes in Dystrophic Muscle [NA]
This clinical trial update discusses biomechanical and morphological changes in dystrophic muscle related to Duchenne muscular dystrophy. Th…
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CLINICAL_TRIALS research · 2017-04-11Clinical Trial Update: Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy [NA]
Clinical trial update analyzing a virtual reality task in patients with Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS research · 2016-08-17Clinical Trial Update: Research of Biomarkers in Duchenne Muscular Dystrophy Patients
This is an update on a clinical trial investigating biomarkers in Duchenne Muscular Dystrophy patients; the study (NCT01380964) sponsored by…
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CLINICAL_TRIALS research · 2016-04-21Clinical Trial Update: Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)
This entry provides an update on a longitudinal natural history study of Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS research · 2016-04-12Clinical Trial Update: Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
This clinical trial update describes an observational study of Duchenne Muscular Dystrophy patients who could theoretically be treated with …
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CLINICAL_TRIALS research · 2015-11-25Clinical Trial Update: The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD [NA]
This entry reports on a clinical trial evaluating remote ischemic preconditioning as a therapy for Duchenne muscular dystrophy, with a focus…
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CLINICAL_TRIALS research · 2015-10-15Clinical Trial Update: Correlation Between Respiratory Impairment and Phonemes Alteration in Dystrophinopathy Patients With Respiratory Failure
This completed clinical trial update documents a study evaluating the correlation between respiratory impairment and phoneme alterations in …
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CLINICAL_TRIALS research · 2015-07-28Clinical Trial Update: Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
An update on the Duchenne Muscular Dystrophy tissue bank for exon skipping (NCT01772043). The current trial status is unknown, and the spons…
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CLINICAL_TRIALS research · 2015-05-07Clinical Trial Update: Upper Limb Assessment in Duchenne Muscular Dystrophy
Clinical trial update on evaluating upper limb function in Duchenne muscular dystrophy; no results reported yet.
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CLINICAL_TRIALS research · 2014-09-17Clinical Trial Update: Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy. [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating autologous bone marrow-derived cells for muscular dystrophy, including Duchenne muscular dystrophy.…
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CLINICAL_TRIALS research · 2007-12-21Clinical Trial Update: Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy [Phase 2]
A completed Phase 2 clinical trial investigated whether oral glutamine can improve muscle mass and function in Duchenne muscular dystrophy.