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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 (1745)

• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Late-Onset Dystrophinopathy. (2021/12/20) ♡
• Identifying the hub genes for Duchenne muscular dystrophy and Becker muscular dystrophy by weighted correlation network analysis. (2021/12/18) ♡
• Synthesis and Exon-Skipping Properties of a 3'-Ursodeoxycholic Acid-Conjugated Oligonucleotide Targeting DMD Pre-mRNA: Pre-Synthetic versus Post-Synthetic Approach. (2021/12/17) ♡
• 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. Pharmacological Profile of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy: A Japanese Experience. (2021/12/16) ♡
• Peptide-Functionalized Dendrimer Nanocarriers for Targeted Microdystrophin Gene Delivery. (2021/12/15) ♡
• Evaluation of a Chair-Mounted Passive Trunk Orthosis: A Pilot Study on Able-Bodied Subjects. (2021/12/15) ♡
• Stabilizing Ryanodine Receptors Improves Left Ventricular Function in Juvenile Dogs With Duchenne Muscular Dystrophy. (2021/12/14) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Ryanodine Receptors Matter for Cardiac Function in Duchenne Muscular Dystrophy: Stability First. (2021/12/14) ♡
• Commentary or editorialiAn expert's opinion or commentary, not new research. Special Issue: Pathophysiology and Therapeutic Perspectives in DMD: The Well-Defined Role of the Immune System. (2021/12/14) ♡
• Haplotype-Based Noninvasive Prenatal Diagnosis of 21 Families With Duchenne Muscular Dystrophy: Real-World Clinical Data in China. (2021/12/14) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Anesthetic management with remimazolam for a pediatric patient with Duchenne muscular dystrophy. (2021/12/10) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. The identification of a novel splicing mutation in the DMD gene of a Chinese family. (2021/12/09) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice. (2021/12/08) ♡
• Reticulon-1C Involvement in Muscle Regeneration. (2021/12/08) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Characterization of Neuromuscular Junctions in Mice by Combined Confocal and Super-Resolution Microscopy. (2021/12/08) ♡
• 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. Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation. (2021/12/07) ♡
• Lifespan Analysis of Dystrophic mdx Fast-Twitch Muscle Morphology and Its Impact on Contractile Function. (2021/12/07) ♡
• Combining Protein Expression and Molecular Data Improves Mutation Characterization of Dystrophinopathies. (2021/12/07) ♡
• Molecular Diagnosis of Muscular Dystrophy Patients in Western Indian Population: A Comprehensive Mutation Analysis Using Amplicon Sequencing. (2021/12/03) ♡
• 7',5'-alpha-bicyclo-DNA: new chemistry for oligonucleotide exon splicing modulation therapy. (2021/12/02) ♡
• A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping. (2021/12/02) ♡
• Profoundly lower muscle mass and rate of contractile protein synthesis in boys with Duchenne muscular dystrophy. (2021/12/01) ♡
• Sensitivity to behavioral stress impacts disease pathogenesis in dystrophin-deficient mice. (2021/12/01) ♡
• Systematic reviewiAll research on one question is searched according to fixed rules and compared side by side, so that no studies with poor outcomes are missed. Antioxidants to prevent respiratory decline in people with Duchenne muscular dystrophy and progressive respiratory decline. (2021/12/01) ♡
• Genetic correction strategies for Duchenne Muscular Dystrophy and their impact on the heart. (2021/12/01) ♡
• Casimersen for Duchenne muscular dystrophy. (2021/12/01) ♡
• 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. Translation of New and Emerging Therapies for Genetic Cardiomyopathies. (2021/12/01) ♡
• Therapeutic Exon Skipping Through a CRISPR-Guided Cytidine Deaminase Rescues Dystrophic Cardiomyopathy in Vivo. (2021/11/30) ♡
• Skeletal Ryanodine Receptors Are Involved in Impaired Myogenic Differentiation in Duchenne Muscular Dystrophy Patients. (2021/11/30) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Severe Combined Immunodeficiency with De Novo Duchenne Muscular Dystrophy Mutation. (2021/11/29) ♡
• Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Co-Occurrence of Fragile X Syndrome with a Second Genetic Condition: Three Independent Cases of Double Diagnosis. (2021/11/27) ♡
• Novel Partial Exon 51 Deletion in the Duchenne Muscular Dystrophy Gene Identified via Whole Exome Sequencing and Long-Read Whole-Genome Sequencing. (2021/11/26) ♡
• Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. (2021/11/24) ♡
• The IRE1/XBP1 signaling axis promotes skeletal muscle regeneration through a cell non-autonomous mechanism. (2021/11/23) ♡
• Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Natural History of a Mouse Model Overexpressing the Dp71 Dystrophin Isoform. (2021/11/23) ♡
• Autonomic Modulation in Duchenne Muscular Dystrophy During a Computer Task: A Prospective Transversal Controlled Trial Assessment by Non-linear Techniques. (2021/11/23) ♡
• Noninvasive prenatal diagnosis of duchenne muscular dystrophy in five Chinese families based on relative mutation dosage approach. (2021/11/22) ♡
• Measuring Duchenne muscular dystrophy impact: development of a proxy-reported measure derived from PROMIS item banks. (2021/11/22) ♡
• β-Dystroglycan Restoration and Pathology Progression in the Dystrophic mdx Mouse: Outcome and Implication of a Clinically Oriented Study with a Novel Oral Dasatinib Formulation. (2021/11/22) ♡
• Retracted publicationiThis research has been withdrawn by science itself, for example due to a flaw in the method or unreliable data. Do not use it as support and do not discuss it as evidence. Optimizing sgRNA to Improve CRISPR/Cas9 Knockout Efficiency: Special Focus on Human and Animal Cell. (2021/11/19) ♡
• An Episomal CRISPR/Cas12a System for Mediating Efficient Gene Editing. (2021/11/18) ♡
• Longitudinal Motor Functional Outcomes and Magnetic Resonance Imaging Patterns of Muscle Involvement in Upper Limbs in Duchenne Muscular Dystrophy. (2021/11/18) ♡
• Implementation of Hospital-Based Supplemental Duchenne Muscular Dystrophy Newborn Screening (sDMDNBS): A Pathway to Broadening Adoption. (2021/11/15) ♡
• Poly C Binding Protein 2 dependent nuclear retention of the utrophin-A mRNA in C2C12 cells. (2021/11/12) ♡
• Incomplete Assembly of the Dystrophin-Associated Protein Complex in 2D and 3D-Cultured Human Induced Pluripotent Stem Cell-Derived Cardiomyocytes. (2021/11/04) ♡
• Alteration of skeletal and cardiac muscles function in DBA/2J mdx mice background: a focus on high intensity interval training. (2021/11/01) ♡
• [Automatic extraction of vertebral landmarks from ultrasound images]. (2021/11/01) ♡
• Efficacy and safety of glucocorticoids in the treatment of progressive muscular dystrophy in children: a systematic review and meta-analysis. (2021/11/01) ♡
• Duchenne Muscular Dystrophy: Genetic and Clinical Profile in the Population of Rajasthan, India. (2021/11/01) ♡
• Orai1-STIM1 Regulates Increased Ca(2+) Mobilization, Leading to Contractile Duchenne Muscular Dystrophy Phenotypes in Patient-Derived Induced Pluripotent Stem Cells. (2021/10/31) ♡

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