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Becker muscular dystrophy
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Publications and studies (1748)
- Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial. (2026/09/08) ♡
- An antisense method for efficient exon skipping and its application to Duchenne muscular dystrophy. (2026/08/18) ♡
- 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. Prevalence and risk factors of bone fragility in duchenne muscular dystrophy: A systematic review and meta-analysis. (2026/08/11) ♡
- Reduced Cx43 S-nitrosylation protects against cardiac stress-induced arrhythmias and myocardial injury in Duchenne Muscular Dystrophy. (2026/08/10) ♡
- Safety, Tolerability, and Efficacy of a Prophylactic Sirolimus Protocol for Patients Receiving Delandistrogene Moxeparvovec-Rokl Gene Therapy. (2026/08/05) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Swallowing Dysfunction and Histopathological Changes in a Rat Model of Duchenne Muscular Dystrophy. (2026/08/04) ♡
- Effects of Virtual Reality Versus Whole-Body Vibration on Muscle Strength and Balance in Children with Duchenne Muscular Dystrophy: a Randomized Controlled Trial. (2026/08/04) ♡
- Evaluation of antibody reactivity to dystrophin products: does Dp40 really exist? (2026/08/04) ♡
- 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. Echocardiography in duchenne muscular dystrophy: a call for consistency and standardisation of reporting. (2026/08/03) ♡
- Randomized researchiParticipants were divided into groups by lottery and compared with each other. This reduces the chance that a difference is due to something other than the treatment. Prednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy. (2026/08/03) ♡
- Sleep Problems in Children With Duchenne Muscular Dystrophy: The Impact on Motor, Cognitive, and Pulmonary Parameters. (2026/08/01) ♡
- Optimizing Care for Growth and Puberty in Duchenne Muscular Dystrophy: A Survey of Clinical Practice in the OPTIMIZE DMD Consortium. (2026/08/01) ♡
- Patient-Reported Symptom Burden in Individuals With Parkinson Disease. (2026/08/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Severe dilated cardiomyopathy in females with dystrophinopathy : a case series of nine patients. (2026/08/01) ♡
- 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. Downstream Pathways of Dystrophin Deficiency in Duchenne Muscular Dystrophy: Implications for Muscle Degeneration and Regeneration. (2026/08/01) ♡
- Engineered exosomes improve myocardial cell membrane integrity and heart function in dystrophic cardiomyopathy. (2026/08/01) ♡
- A high-fat, high-sucrose diet exacerbates muscle and metabolic pathology and undermines glucocorticoid efficacy in dystrophin-deficient mice. (2026/08/01) ♡
- 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. Nutritional interventions and dietary supplements in muscle diseases: a systematic review. (2026/08/01) ♡
- Improving Nocturnal Non-Invasive Positive Pressure Support Adherence in Children and Young Adults With Duchenne Muscular Dystrophy. (2026/08/01) ♡
- Use of SGLT2 Inhibitors in Dystrophin Deficient Cardiomyopathy: A Multi-center Study Using the ACTION Registry. (2026/08/01) ♡
- 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. Long-read sequencing for neurological disorders: opportunities, challenges, and future directions. (2026/08/01) ♡
- Gamified occupational therapy program for adolescents with Duchenne muscular dystrophy: A study protocol for randomized controlled trial. (2026/07/30) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Inpatient rehabilitation admission for a patient with Duchenne muscular dystrophy status post Achilles tendon release. (2026/07/29) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Role of Finerenone in Duchenne Muscular Dystrophy in a 45-Year-Old Man: Outcome and Brief Review. (2026/07/29) ♡
- Deramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial. (2026/07/29) ♡
- 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. Cytoskeleton-Membrane Uncoupling in Duchenne Muscular Dystrophy: Implications for Newborn Screening and Early Protection. (2026/07/29) ♡
- The Subclinical Window for Cardioprotection in Duchenne Muscular Dystrophy: A Systematic Review and Meta-analysis. (2026/07/28) ♡
- Niclosamide reprograms macrophages and improves muscle integrity in Duchenne muscular dystrophy models. (2026/07/28) ♡
- Seventeen-year follow-up of hypophosphatasia diagnosed in middle-aged siblings harboring a novel intronic and a rare missense ALPL gene mutation. (2026/07/28) ♡
- Effects of intermittent corticosteroids on scoliosis, bone density, and vertebral fractures in duchenne muscular dystrophy. (2026/07/24) ♡
- Characterizing obsessive-compulsive features in Duchenne muscular dystrophy: a DSM-5 approach. (2026/07/23) ♡
- Evaluating exon skipping in the central nervous system in Duchenne muscular dystrophy using spatial transcriptomics. (2026/07/23) ♡
- 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. Accuracy of the molecular diagnosis of duchenne and becker muscular dystrophy: A systematic review with meta-analysis. (2026/07/21) ♡
- 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. Clinical Utility and Measurement Properties of Balance Measures in Duchenne Muscular Dystrophy: A Scoping Review. (2026/07/21) ♡
- Beyond Teleconsultation: Exploring the Role of Mobile Health Technologies in Duchenne Muscular Dystrophy. (2026/07/21) ♡
- The Impact of Clinical Trial Participation on Quality of Life in Duchenne Muscular Dystrophy. (2026/07/20) ♡
- Myocardial fat fraction in Becker muscular dystrophy and women carrying pathogenic DMD gene variants assessed by Dixon cardiac MRI. (2026/07/20) ♡
- 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. Recommendations for the use of clinical outcome assessments in rare disease drug development. (2026/07/20) ♡
- Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy: A Critical Synthesis of Approved and Emerging Strategies Through the Lens of an Underrepresented Population. (2026/07/20) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Clinical challenges of an Xp21 contiguous gene deletion syndrome in a newborn and 15 months of follow-up - case report. (2026/07/20) ♡
- 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. When barriers fail: the role of endothelial dysfunction in rare pediatric neuromuscular diseases. (2026/07/20) ♡
- Clinical spectrum and ascertainment pathways in pediatric female dystrophinopathy. (2026/07/18) ♡
- 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 future of pediatric gene therapy: CRISPR-Cas9, AI, and personalized medicine. (2026/07/17) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy. (2026/07/17) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Dysregulation of the Schwann cell GABAergic system and Aβ-fibers conduction abnormalities in the sciatic nerve of the mdx mouse model of Duchenne muscular dystrophy. (2026/07/16) ♡
- 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. Impaired Myogenic Differentiation Is a Shared Feature Across Genetic Myopathies. (2026/07/16) ♡
- Prevalence of Depression among Children with Duchenne Muscular Dystrophy: A Cross-sectional Survey Study. (2026/07/15) ♡
- 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. Mechanisms of Impaired Skeletal Muscle Regeneration and Therapeutic Approaches in Aging and Chronic Disease. (2026/07/15) ♡
- Expanding Vamorolone Treatment Access for Canadians with Duchenne Muscular Dystrophy. (2026/07/13) ♡
- Estimating Utility Values Using the DMD-QoL: A Disease-Specific Preference-Based Measure for Duchenne Muscular Dystrophy (DMD). (2026/07/13) ♡
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