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Duchenne muscular dystrophy

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Search Period All yearsLast 12 monthsLast 5 years Topic All topicsGeneralNutrition and dietAlternative treatment methods Type of research Any type of researchHighest level of evidence only (36)Meta-analysis (9)Randomized trial (11)Systematic review (9)Phase 3 study (7)Clinical examination (506)Review article (205)Early study (phase 1 or 2) (27)Case report (101)Laboratory or animal study (64)Commentary or editorial (52)Retracted Publication (4) Studies Currently Seeking Participants Only (38)

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Publications and studies (1744)

• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) (2020-08-04) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) (2020-07-29) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) (2020-07-15) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Non-invasive Molecular Imaging of Muscle Structure (MSOT_muscles) (2020-07-07) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Digestive Events in Duchenne Muscular Dystrophy Patients (2020-05-12) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy (2020-04-20) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. European Home Mechanical Ventilation Registry (2020-04-09) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) (2020-04-07) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy (2020-03-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients (2020-03-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy (2020-03-30) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. CRT-P or CRT-D in Dilated Cardiomyopathy (2020-03-25) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy (2020-03-17) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Evaluation of Muscle Activation in Climbing up Stairs Activity in Children With Duchenne Muscular Dystrophy (2020-02-27) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Exploratory Study of NS-065/NCNP-01 in DMD (2020-02-26) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy (2020-02-07) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy (2020-01-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy (2020-01-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Tadalafil and Sildenafil for Duchenne Muscular Dystrophy (2020-01-14) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Sodium Nitrate for Muscular Dystrophy (2020-01-13) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD) (2020-01-02) ♡
• T cell receptor specificity drives accumulation of a reparative population of regulatory T cells within acutely injured skeletal muscle. (2019/12/26) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Inhibition of FLT1 ameliorates muscular dystrophy phenotype by increased vasculature in a mouse model of Duchenne muscular dystrophy. (2019/12/26) ♡
• S-nitrosylation of connexin43 hemichannels elicits cardiac stress-induced arrhythmias in Duchenne muscular dystrophy mice. (2019/12/19) ♡
• Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Pulsed glucocorticoids enhance dystrophic muscle performance through epigenetic-metabolic reprogramming. (2019/12/19) ♡
• An improved method for studying mouse diaphragm function. (2019/12/19) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. S1P/S1P Receptor Signaling in Neuromuscular Disorders. (2019/12/17) ♡
• Use of air stacking to improve pulmonary function in Indonesian Duchenne muscular dystrophy patients: bridging the standard of care gap in low middle income country setting. (2019/12/16) ♡
• Development of a high-throughput screen to identify small molecule enhancers of sarcospan for the treatment of Duchenne muscular dystrophy. (2019/12/12) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. In vivo cerebellar circuit function is disrupted in an mdx mouse model of Duchenne muscular dystrophy. (2019/12/09) ♡
• Exploring physiological signals on people with Duchenne muscular dystrophy for an active trunk support: a case study. (2019/12/09) ♡
• Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models. (2019/12/06) ♡
• Meta-analysisiAll studies on one question combined and calculated together. This is the strongest form of research that exists: a single loose study can be coincidence, dozens together much less so. The label says something about the design, not about the outcome — which can also be that something does NOT work. Strength training and aerobic exercise training for muscle disease. (2019/12/06) ♡
• CRISPR-Cas3 induces broad and unidirectional genome editing in human cells. (2019/12/06) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Recurrent hypotension induced by sacubitril/valsartan in cardiomyopathy secondary to Duchenne muscular dystrophy: A case report. (2019/12/06) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Metabolic Alterations in Cardiomyocytes of Patients with Duchenne and Becker Muscular Dystrophies. (2019/12/05) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Characterization of mesoangioblast cell fate and improved promyogenic potential of a satellite cell-like subpopulation upon transplantation in dystrophic murine muscles. (2019/12/01) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Importance of muscle biopsy to establish pathogenicity of DMD missense and splice variants. (2019/12/01) ♡
• Psychiatric and neurodevelopmental aspects of Becker muscular dystrophy. (2019/12/01) ♡
• Evaluation of gait in Duchenne Muscular Dystrophy: Relation of 3D gait analysis to clinical assessment. (2019/12/01) ♡
• Detection of collagens by multispectral optoacoustic tomography as an imaging biomarker for Duchenne muscular dystrophy. (2019/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. The Dystrophinopathies. (2019/12/01) ♡
• The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy. (2019/12/01) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Gene Therapy Briefs. (2019/12/01) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Muscular dystrophies. (2019/11/30) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Teaching an Old Molecule New Tricks: Drug Repositioning for Duchenne Muscular Dystrophy. (2019/11/30) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. QuantiMus: A Machine Learning-Based Approach for High Precision Analysis of Skeletal Muscle Morphology. (2019/11/29) ♡
• Review articleiA summary of what is known about a topic, written by experts. Not compiled according to fixed search rules, so the selection of studies may be biased. Biomaterials and Advanced Biofabrication Techniques in hiPSCs Based Neuromyopathic Disease Modeling. (2019/11/29) ♡
• Micro-dystrophin Gene Therapy Partially Enhances Exercise Capacity in Older Adult mdx Mice. (2019/11/27) ♡
• N-acetylcysteine Decreases Fibrosis and Increases Force-Generating Capacity of mdx Diaphragm. (2019/11/24) ♡

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