Becker muscular dystrophy
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Publications and studies (1748)
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Eteplirsen Use in a Boy with Duchenne Muscular Dystrophy and Sickle Cell Anemia. (2022/10/24) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD. (2022/10/23) ♡
- Single cell sequencing maps skeletal muscle cellular diversity as disease severity increases in dystrophic mouse models. (2022/10/21) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Low-Intensity Vibration Protects the Weight-Bearing Skeleton and Suppresses Fracture Incidence in Boys With Duchenne Muscular Dystrophy: A Prospective, Randomized, Double-Blind, Placebo-Controlled Clinical Trial. (2022/10/18) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Perks of Rehabilitation in Improving Motor Function in a Nine-Year-Old Male With Duchenne Muscular Dystrophy: A Case Report. (2022/10/11) ♡
- The association between dietary factors and body weight and composition in boys with Duchenne muscular dystrophy. (2022/10/01) ♡
- Structure-activity relationship study of PROTACs against hematopoietic prostaglandin D(2) synthase. (2022/09/23) ♡
- The skeletal muscle phenotype of the DE50-MD dog model of Duchenne muscular dystrophy. (2022/09/23) ♡
- Development of clinical practice guidelines for allied health and nursing assessment and management of Duchenne muscular dystrophy. (2022/09/01) ♡
- Evaluation of the DBA/2J mouse as a potential background strain for genetic models of cardiomyopathy. (2022/09/01) ♡
- Longitudinal assessment of blood-borne musculoskeletal disease biomarkers in the DE50-MD dog model of Duchenne muscular dystrophy. (2022/08/17) ♡
- The Relationship between Obesity and Clinical Outcomes in Young People with Duchenne Muscular Dystrophy. (2022/08/12) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Responsiveness and Minimal Clinically Important Difference of the Chinese Version of the Motor Function Measure-32 in Children and Adolescents with Duchenne Muscular Dystrophy. (2022/08/01) ♡
- 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. MicroRNAs in Dystrophinopathy. (2022/07/14) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. A medium-chain triglyceride containing ketogenic diet exacerbates cardiomyopathy in a CRISPR/Cas9 gene-edited rat model with Duchenne muscular dystrophy. (2022/07/08) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Morbidity and mortality associated with gastrointestinal dysfunction in neuromuscular disease: a single-centre case series. (2022/07/01) ♡
- Cardio-respiratory and phenotypic rescue of dystrophin/utrophin-deficient mice by combination therapy. (2022/06/07) ♡
- Iron overload and impaired iron handling contribute to the dystrophic pathology in models of Duchenne muscular dystrophy. (2022/06/01) ♡
- Leveraging cardiac magnetic resonance imaging to assess skeletal muscle progression in Duchenne muscular dystrophy. (2022/05/01) ♡
- Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies. (2022/04/01) ♡
- 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. The impact of genotype on outcomes in individuals with Duchenne muscular dystrophy: A systematic review. (2022/03/01) ♡
- Performance of Passive Muscle Stiffness in Diagnosis and Assessment of Disease Progression in Duchenne Muscular Dystrophy. (2022/03/01) ♡
- Early-stage study (phase 1 or 2)iEarly research in a small group, primarily aimed at safety and dosage. Whether it actually works still needs to be determined afterward. Characterization of patients with Becker muscular dystrophy by histology, magnetic resonance imaging, function, and strength assessments. (2022/03/01) ♡
- 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. Pro-inflammatory monocytes are increased in Duchenne muscular dystrophy and suppressed with omega-3 fatty acids: A double-blind, randomized, placebo-controlled pilot study. (2022/03/01) ♡
- Improving Access and Guideline Adherence in Pulmonary Care in Patients With Duchenne Muscular Dystrophy. (2022/03/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. BMI-z scores of boys with Duchenne muscular dystrophy already begin to increase before losing ambulation: a longitudinal exploration of BMI, corticosteroids and caloric intake. (2022/03/01) ♡
- Natural History of Histopathologic Changes in Cardiomyopathy of Golden Retriever Muscular Dystrophy. (2022/02/17) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Phenotypic Spectrum of Dystrophinopathy Due to Duchenne Muscular Dystrophy Exon 2 Duplications. (2022/02/15) ♡
- Hepatic Steatosis Assessment as a New Strategy for the Metabolic and Nutritional Management of Duchenne Muscular Dystrophy. (2022/02/09) ♡
- Benefits of Telemonitoring of Pulmonary Function-3-Month Follow-Up of Home Electronic Spirometry in Patients with Duchenne Muscular Dystrophy. (2022/02/06) ♡
- 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. CRISPR-Cas9-Mediated Gene Therapy in Neurological Disorders. (2022/02/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Authors' Response to Comment on "Assessing the Appropriateness of the EQ-5D for Duchenne Muscular Dystrophy: A Patient-Centered Study". (2022/02/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Measuring What Matters: Little Evidence Supporting the Content Validity of EQ-5D in People with Duchenne Muscular Dystrophy and Their Caregivers. (2022/02/01) ♡
- 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 prehabilitation in pediatric anesthesia in children with muscular and neurologic disease. (2022/02/01) ♡
- 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. Histopathology of Duchenne muscular dystrophy in correlation with changes in proteomic biomarkers. (2022/02/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. A 'virtual' revolution: non-invasive methods to probe skeletal muscle metabolism in Duchenne muscular dystrophy. (2022/02/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Comparing contributors of parental sleep health in families with and without a child with Duchenne muscular dystrophy. (2022/02/01) ♡
- Psychosocial adjustment in adults with Duchenne muscular dystrophy: A pilot study on a shortened parent-report questionnaire. (2022/02/01) ♡
- Cholesterol absorption blocker ezetimibe prevents muscle wasting in severe dysferlin-deficient and mdx mice. (2022/02/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Growth pattern trajectories in boys with Duchenne muscular dystrophy. (2022/01/24) ♡
- 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. Anti-Remodeling Cardiac Therapy in Patients With Duchenne Muscular Dystrophy, Meta-Analysis Study. (2022/01/20) ♡
- 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. Dystrophinopathies. (2022/01/20) ♡
- 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. Therapeutic approaches to preserve the musculature in Duchenne Muscular Dystrophy: The importance of the secondary therapies. (2022/01/15) ♡
- Lipidomic Analyses Reveal Specific Alterations of Phosphatidylcholine in Dystrophic Mdx Muscle. (2022/01/12) ♡
- Corrigendum: Muscle Twitch Kinetics Are Dependent on Muscle Group, Disease State, and Age in Duchenne Muscular Dystrophy Mouse Models. (2022/01/05) ♡
- 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. Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence. (2022/01/05) ♡
- Use of a book to disclose a diagnosis of Duchenne muscular dystrophy to a young child: A pilot study. (2022/01/01) ♡
- KLF15 overexpression in myocytes fails to ameliorate ALS-related pathology or extend the lifespan of SOD1G93A mice. (2022/01/01) ♡
- 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. Assessment of left ventricular dyssynchrony by speckle tracking echocardiography in children with duchenne muscular dystrophy. (2022/01/01) ♡
- 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. Differential fates of introns in gene expression due to global alternative splicing. (2022/01/01) ♡
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