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Becker muscular dystrophy
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Publications and studies (1748)
- 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. AMONDYS 45 (Casimersen), a Novel Antisense Phosphorodiamidate Morpholino Oligomer: Clinical Considerations for Treatment in Duchenne Muscular Dystrophy. (2023/12/28) ♡
- Establishment of a Triple Quadrupole HPLC-MS Quantitation Method for Dystrophin Protein in Mouse and Human Skeletal Muscle. (2023/12/25) ♡
- Astaxanthin Ameliorates Worsened Muscle Dysfunction of MDX Mice Fed with a High-Fat Diet through Reducing Lipotoxicity and Regulating Gut Microbiota. (2023/12/21) ♡
- mCherry on Top: A Positive Read-Out Cellular Platform for Screening DMD Exon Skipping Xenopeptide-PMO Conjugates. (2023/12/20) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Efficacy of N-163 beta-glucan in beneficially improving biomarkers of relevance to muscle function in patients with muscular dystrophies in a pilot clinical study. (2023/12/20) ♡
- Estimating health state utilities in Duchenne muscular dystrophy using the health utilities index and EQ-5D-5L. (2023/12/15) ♡
- A qualitative study to understand the Duchenne muscular dystrophy experience from the parent/patient perspective. (2023/12/12) ♡
- Derivation and validation of diagnostic models for myocardial fibrosis in duchenne muscular dystrophy: assessed by multi-parameter cardiovascular magnetic resonance. (2023/12/11) ♡
- 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. Evaluation of Chemically Modified Nucleic Acid Analogues for Splice Switching Application. (2023/12/11) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Safety concerns surrounding AAV and CRISPR therapies in neuromuscular treatment. (2023/12/08) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. (2023/12/07) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. (2023/12/07) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. Reply. (2023/12/07) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model. (2023/12/07) ♡
- Mutational spectrum and phenotypic variability of Duchenne muscular dystrophy and related disorders in a Bangladeshi population. (2023/12/06) ♡
- Replenishing NAD(+) content reduces aspects of striated muscle disease in a dog model of Duchenne muscular dystrophy. (2023/12/04) ♡
- Development of Conformationally Restricted Negamycin Derivatives for Potent Readthrough Activity. (2023/12/04) ♡
- A fat- and sucrose-enriched diet causes metabolic alterations in mdx mice. (2023/12/01) ♡
- Appendicular lean mass index changes in patients with Duchenne muscular dystrophy and Becker muscular dystrophy. (2023/12/01) ♡
- Ryanodine receptor type 1 content decrease-induced endoplasmic reticulum stress is a hallmark of myopathies. (2023/12/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Impact of the Inhibition of Organic Anion Transporter on Tricyclo-DNA-Mediated Exon Skipping in the mdx Mouse Model. (2023/12/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy. (2023/12/01) ♡
- Prenatal diagnosis of 1408 foetuses at risk of DMD/BMD by MLPA and Sanger sequencing combined with STR linkage analysis. (2023/12/01) ♡
- Longitudinal assessment of skeletal muscle functional mechanics in the DE50-MD dog model of Duchenne muscular dystrophy. (2023/12/01) ♡
- Patient-Reported Impact of Symptoms in Spinal and Bulbar Muscular Atrophy. (2023/12/01) ♡
- Efficient exon skipping by base-editor-mediated abrogation of exonic splicing enhancers. (2023/11/28) ♡
- A 1-year analysis from a natural history study in Chinese individuals with Duchenne muscular dystrophy. (2023/11/28) ♡
- Assessment of Motor Unit Potentials Duration as the Biomarker of DT-DEC01 Cell Therapy Efficacy in Duchenne Muscular Dystrophy Patients up to 12 Months After Systemic-Intraosseous Administration. (2023/11/24) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. A novel splicing mutation identified in a DMD patient: a case report. (2023/11/20) ♡
- Electrical impedance myography detects dystrophin-related muscle changes in mdx mice. (2023/11/18) ♡
- Mutation spectrum analysis of DMD gene in Indonesian Duchenne and Becker muscular dystrophy patients. (2023/11/17) ♡
- Indications, types, and diagnostic implications of prenatal genetic testing in Sub-Saharan Africa: A descriptive study. (2023/11/16) ♡
- 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. Mental health challenges and digital platform opportunities in patients and families affected by pediatric neuromuscular diseases - experiences from Switzerland. (2023/11/16) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Editorial: Personalized medicine for neuromuscular disorders. (2023/11/16) ♡
- Confirmatory validation of the french version of the Duchenne Muscular Dystrophy module of the pediatric quality of life inventory (PedsQL(TM)3.0DMDfv). (2023/11/15) ♡
- Effectiveness of a 5-Week Virtual Reality Telerehabilitation Program for Children With Duchenne and Becker Muscular Dystrophy: Prospective Quasi-Experimental Study. (2023/11/15) ♡
- Proteome Profiling of the Dystrophic mdx Mice Diaphragm. (2023/11/13) ♡
- 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. Detecting early signs in Duchenne muscular dystrophy: comprehensive review and diagnostic implications. (2023/11/10) ♡
- Regional 4D Cardiac Magnetic Resonance Strain Predicts Cardiomyopathy Progression in Duchenne Muscular Dystrophy. (2023/11/08) ♡
- Myospreader improves gene editing in skeletal muscle by myonuclear propagation. (2023/11/06) ♡
- Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy. (2023/11/02) ♡
- Factors influencing the decision to introduce alternative nutrition in patients with Duchenne muscular dystrophy. (2023/11/01) ♡
- Life-threatening bowel complications in adults with Duchenne muscular dystrophy: a case series. (2023/11/01) ♡
- 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. A systematic literature review and meta-analysis of the effectiveness of vitamin D supplementation for patients with Duchenne muscular dystrophy. (2023/11/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Cryptic exon activation caused by a novel deep-intronic splice-altering variant in Becker muscular dystrophy. (2023/11/01) ♡
- [Microtubular network and functionality of the striated skeletal muscle]. (2023/11/01) ♡
- 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. Gene therapy for primary myopathies: literature review and prospects. (2023/11/01) ♡
- 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. Basic notions about gene therapy from the nucleic acid perspective and applications in a pediatric disease: Duchenne muscular dystrophy. (2023/11/01) ♡
- Uridine Administration Promotes Normalization of Heart Mitochondrial Function in Dystrophin-Deficient Mice and Decreases Tissue Fibrosis. (2023/11/01) ♡
- Higher Prevalence of Nonsense Pathogenic DMD Variants in a Single-Center Cohort from Brazil: A Genetic Profile Study That May Guide the Choice of Disease-Modifying Treatments. (2023/10/28) ♡
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