Duchenne muscular dystrophy
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Publications (1249)
- (1) H NMRS of carnosine combined with (31) P NMRS to better characterize skeletal muscle pH dysregulation in Duchenne muscular dystrophy. (2018/01/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Early involvement of the supinator muscle in Duchenne muscular dystrophy. (2018/01/01) ♡
- Clinical trialiResearch in patients, without randomization between groups. Useful, but less certain than a randomized trial. Timed function tests, motor function measure, and quantitative thigh muscle MRI in ambulant children with Duchenne muscular dystrophy: A cross-sectional analysis. (2018/01/01) ♡
- Diagnostic and clinical significance of the titin fragment in urine of Duchenne muscular dystrophy patients. (2018/01/01) ♡
- 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. Antisense oligonucleotides: the next frontier for treatment of neurological disorders. (2018/01/01) ♡
- Phase 3 studyiResearch in a large group of patients, the final step before a treatment can be approved. What comes out here carries significant weight. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy. (2018/01/01) ♡
- 226(th) ENMC International Workshop:: Towards validated and qualified biomarkers for therapy development for Duchenne muscular dystrophy 20-22 January 2017, Heemskerk, The Netherlands. (2018/01/01) ♡
- Correlating motor unit morphology with bioelectrical activity - A simulation study. (2018/01/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Biomarkers of Cardiac Disease in Duchenne Muscular Dystrophy are Insufficient in the Absence of Clinical Cardiologic and Neurologic Assessment. (2018/01/01) ♡
- ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs. (2018/01/01) ♡
- Altered myofilament structure and function in dogs with Duchenne muscular dystrophy cardiomyopathy. (2018/01/01) ♡
- Effective regeneration of dystrophic muscle using autologous iPSC-derived progenitors with CRISPR-Cas9 mediated precise correction. (2018/01/01) ♡
- 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. Subcellular Targeting of Nitric Oxide Synthases Mediated by Their N-Terminal Motifs. (2018/01/01) ♡
- 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. Basics of bone metabolism and osteoporosis in common pediatric neuromuscular disabilities. (2018/01/01) ♡
- α-Ketoglutarate prevents skeletal muscle protein degradation and muscle atrophy through PHD3/ADRB2 pathway. (2018/01/01) ♡
- 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. Disturbed Ca(2+) Homeostasis in Muscle-Wasting Disorders. (2018/01/01) ♡
- 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. Human iPSC Models to Study Orphan Diseases: Muscular Dystrophies. (2018/01/01) ♡
- 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. Review of Recent Patents and Developments in Skeletal Muscle Regeneration. (2018/01/01) ♡
- A Simple and Low-cost Assay for Measuring Ambulation in Mouse Models of Muscular Dystrophy. (2017/12/29) ♡
- 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. Role of transforming growth factor-β in muscle damage and regeneration: focused on eccentric muscle contraction. (2017/12/27) ♡
- Low-intensity training provokes adaptive extracellular matrix turnover of a muscular dystrophy model. (2017/12/27) ♡
- Epigenetic Regulators Modulate Muscle Damage in Duchenne Muscular Dystrophy Model. (2017/12/21) ♡
- Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne. (2017/12/20) ♡
- Echocardiographic Image Quality Deteriorates with Age in Children and Young Adults with Duchenne Muscular Dystrophy. (2017/12/20) ♡
- Increased Number of Circulating CD8/CD26 T Cells in the Blood of Duchenne Muscular Dystrophy Patients Is Associated with Augmented Binding of Adenosine Deaminase and Higher Muscular Strength Scores. (2017/12/18) ♡
- Rational Design of Short Locked Nucleic Acid-Modified 2'-O-Methyl Antisense Oligonucleotides for Efficient Exon-Skipping In Vitro. (2017/12/15) ♡
- Link between MHC Fiber Type and Restoration of Dystrophin Expression and Key Components of the DAPC by Tricyclo-DNA-Mediated Exon Skipping. (2017/12/15) ♡
- In Vivo Target Gene Activation via CRISPR/Cas9-Mediated Trans-epigenetic Modulation. (2017/12/14) ♡
- Transient receptor potential channel 6 regulates abnormal cardiac S-nitrosylation in Duchenne muscular dystrophy. (2017/12/12) ♡
- CRISPR/Cas9 delivery with one single adenoviral vector devoid of all viral genes. (2017/12/07) ♡
- Pharmacological inhibition of REV-ERB stimulates differentiation, inhibits turnover and reduces fibrosis in dystrophic muscle. (2017/12/07) ♡
- Tempol Supplementation Restores Diaphragm Force and Metabolic Enzyme Activities in mdx Mice. (2017/12/06) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Glucocorticoids Improve Myogenic Differentiation In Vitro by Suppressing the Synthesis of Versican, a Transitional Matrix Protein Overexpressed in Dystrophic Skeletal Muscles. (2017/12/06) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Derivation of the Duchenne muscular dystrophy patient-derived induced pluripotent stem cell line lacking DMD exons 49 and 50 (CCMi001DMD-A-3, ∆49, ∆50). (2017/12/01) ♡
- Interleukin-1beta (IL-1β)-induced Notch ligand Jagged1 suppresses mitogenic action of IL-1β on human dystrophic myogenic cells. (2017/12/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Therapeutic Options to Improve Bone Health Outcomes in Duchenne Muscular Dystrophy: Zoledronic Acid and Pubertal Induction. (2017/12/01) ♡
- Immunoglobulin therapy ameliorates the phenotype and increases lifespan in the severely affected dystrophin-utrophin double knockout mice. (2017/12/01) ♡
- Description of individual patientsiThe story of one or a couple of patients. Informative, but you cannot infer from it whether something works in general. Prenatal diagnosis for a Chinese family with a de novo DMD gene mutation: A case report. (2017/12/01) ♡
- Laboratory or animal researchiNo research in humans yet. Promising in a test tube or in mice unfortunately means nothing for patients. Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy. (2017/11/29) ♡
- Evaluation of cardiac functions in children with Duchenne Muscular Dystrophy: A prospective case-control study. (2017/11/25) ♡
- Abnormal carbohydrate metabolism in a canine model for muscular dystrophy. (2017/11/23) ♡
- Muscle MRI and functional outcome measures in Becker muscular dystrophy. (2017/11/22) ♡
- Differences in vertebral morphology around the apical vertebrae between neuromuscular scoliosis and idiopathic scoliosis in skeletally immature patients: a three-dimensional morphometric analysis. (2017/11/16) ♡
- Are mice good models for human neuromuscular disease? Comparing muscle excursions in walking between mice and humans. (2017/11/16) ♡
- The primary cilium is necessary for the differentiation and the maintenance of human adipose progenitors into myofibroblasts. (2017/11/10) ♡
- Vitamin E treatment decreases muscle injury in mdx mice. (2017/11/01) ♡
- Commentary or editorialiAn expert's opinion or commentary, not new research. Creatine kinase: how an obsolete test for skeletal muscle disease became a risk factor for hypertension. (2017/11/01) ♡
- Clinical profiles and prognosis of acute heart failure in adult patients with dystrophinopathies on home mechanical ventilation. (2017/11/01) ♡
- 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. Regulation of Skeletal Muscle Plasticity by Protein Arginine Methyltransferases and Their Potential Roles in Neuromuscular Disorders. (2017/11/01) ♡
- Real-World Evidence, Public Participation, and the FDA. (2017/11/01) ♡
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