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Duchenne muscular dystrophy
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Publications and studies (1744)
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Investigation of the Relationship Between Executive Functions and Occupational Performance of Children With Duchenne Muscular Dystrophy (2026-02-23) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Trial to Investigate Safety and Pharmacokinetics of GRT6019, Including Food Effect, in Healthy Male Participants (2026-02-13) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Natural History of Duchenne Muscular Dystrophy (2026-02-12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Registry for Duchenne and Becker Muscular Dystrophy (2026-02-11) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (2026-02-06) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Factors Influencing Physical Activity Levels in Children With Duchenne Muscular Dystrophy: An ICF-CY-Based Study (2026-01-29) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy (2026-01-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. DMD and Gamified Physiotherapy (2026-01-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. The Effect of Dual-tasking Program on Cognitive and Physical Functions and Independence in Activities of Daily Living in Children With Duchenne Muscular Dystrophy: A Single-blind Randomized Controlled Trial (2026-01-27) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children With DMD/SMA (2026-01-22) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study (2026-01-21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy (2026-01-20) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias (2026-01-16) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. A Study to Evaluate the Safety and Tolerability of RAG-18 in Pediatric Patients With Duchenne Muscular Dystrophy (2026-01-16) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Virtual Reality and Whole Body Vibration on Muscle Strength and Balance in Children With DMD (2026-01-14) ♡
- Inhibition of HuR/ELAVL-1 attenuates fibrotic progression in Mdx mice with dilated cardiomyopathy. (2025/12/30) ♡
- Evaluation of a control paradigm allowing heart rate guided rehabilitative exercise for boys with Duchenne muscular dystrophy. (2025/12/29) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. PPMO-based exon skipping therapy improves respiratory function in the mdx mouse model of Duchenne muscular dystrophy. (2025/12/26) ♡
- Statistical Genetics of DMD Gene Mutations in a Kazakhstan Cohort: MLPA/NGS Variant Validation and Genotype-Phenotype Modelling. (2025/12/26) ♡
- Inhibition of TRPC3-Nox2 Complex Formation Ameliorates Skeletal Muscle Atrophy. (2025/12/26) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A Rare Co-occurrence of Duchenne Muscular Dystrophy and Glycerol Kinase Deficiency Associated With Xp21 Contiguous Gene Deletion Syndrome: A Case Report. (2025/12/25) ♡
- Validation of the Japanese Version of the Muscular Dystrophy Spine Questionnaire in Patients with Flaccid Neuromuscular Scoliosis in Japan. (2025/12/25) ♡
- Spectrum of Osteoporosis Etiologies with Associated Vertebral Compression Fractures in Children: Analysis of 11 Cases. (2025/12/24) ♡
- Brain Matters in Duchenne Muscular Dystrophy: DMD Mutation Sites and Their Association with Neurological Comorbidities Through Isoform Impairment. (2025/12/24) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Symptomatic Pneumoperitoneum After Percutaneous Radiological Gastrostomy in Patient With Duchenne Disease Dependent on Non-Invasive Mechanical Ventilation. (2025/12/24) ♡
- Morpholino-RNA duplex exhibits robust, sustained, and safe steric-block antisense activity by intracerebroventricular and intrathecal injection. (2025/12/22) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Validation of an in vitro muscle platform to evaluate myogenesis and calcium handling in control and dystrophic human myotubes. (2025/12/17) ♡
- Development of a cost-effectiveness analysis model for Duchenne muscular dystrophy utilizing the national registry in Japan. (2025/12/17) ♡
- 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 Promise and Pitfalls of AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy. (2025/12/17) ♡
- 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. Interplay between mTORC1 signaling and iron homeostasis in muscle atrophy. (2025/12/16) ♡
- Assessing Delays in Time to Diagnosis of Duchenne Muscular Dystrophy: A Survey of Current Primary Care Practices. (2025/12/15) ♡
- Upper limb function and quality of life in Duchenne muscular dystrophy: a cross-sectional study in Chile. (2025/12/12) ♡
- 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. Antisense and gene therapy trials in Duchenne muscular dystrophy. (2025/12/12) ♡
- 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. Beginning of a new era of synthetic messenger RNA therapeutics: Comprehensive insights on mRNA drug design, development and applications. (2025/12/12) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Transcytotic transportation of size-controlled nanocarriers into dystrophic skeletal muscle leads to therapeutic outcome in mice. (2025/12/11) ♡
- Tonic GABA(A) receptor currents in Cerebellar Purkinje cells of wild-type and DMD(mdx) mice. (2025/12/11) ♡
- Optical genome mapping identifies a balanced inversion disrupting DMD in a patient with Duchenne muscular dystrophy. (2025/12/07) ♡
- Duchenne Muscular Dystrophy in the Republic of North Ossetia-Alania: Epidemiological Study, Diagnostic Issues, and Treatment Prospects. (2025/12/06) ♡
- 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. A review of recent studies on CRISPR/Cas9-mediated genome editing in a variety of muscle-related genetic disorders. (2025/12/05) ♡
- Endocrine and metabolic complications in a national cohort of Slovene children and adolescents with Duchenne muscular dystrophy: real-world criteria for transition to vamorolone therapy. (2025/12/05) ♡
- 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. Histone deacetylases in Duchenne muscular dystrophy: a role in the mechanism of disease and a target for inhibition. (2025/12/04) ♡
- Conditional Dmd ablation in muscle and brain causes profound effects on muscle function and neurobehavior. (2025/12/02) ♡
- Comprehensive Profiling of Annexins in Neuromuscular Disorders Reveals a Unique Signature in Dysferlinopathy. (2025/12/01) ♡
- [Duchenne de Boulogne: Pioneer of Neurology]. (2025/12/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Duchenne muscular dystrophy in 2025. (2025/12/01) ♡
- Duchenne muscular dystrophy: the French Dystrophinopathies Registry (DYS Registry). (2025/12/01) ♡
- 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. Multidisciplinary management of Duchenne muscular dystrophy from childhood to adulthood. (2025/12/01) ♡
- 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. Cardiac care in Duchenne muscular dystrophy. (2025/12/01) ♡
- 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. Respiratory care in Duchenne muscular dystrophy. (2025/12/01) ♡
- 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. Genetics and pathophysiology of Duchenne muscular dystrophy. (2025/12/01) ♡
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