What researchers published on Duchenne muscular dystrophy
736 papers, newest first.
Showing 100 of 736
Longitudinal pulmonary outcomes after posterior spinal fusion in Duchenne muscular dystrophy: a retrospective cohort study.European journal of orthopaedic surgery & traumatology : orthopedie traumatologie
ReviewNewborn Screening for Duchenne Muscular Dystrophy: Contemporary Considerations for Implementation of an Early Diagnostic Paradigm.Neurology
Advancing Clinical Trials and Precision Medicine in Duchenne Muscular Dystrophy Using Machine Learning-Based Trajectory Modeling and MRI Radiomics.Clinical pharmacology and therapeutics
Neurochemical signatures of morphometric network reorganization in Duchenne muscular dystrophy: Links to cognition and behavior.Progress in neuro-psychopharmacology & biological psychiatry
Case reportA late-onset phenotype of familial Becker muscular dystrophy with DMD exon 26-34 deletion: a case report.BMC neurology
The prevalence of Duchenne/Becker muscular dystrophy in the Russian Federation.Frontiers in genetics
Case reportDelayed Recognition of Duchenne Muscular Dystrophy During an Acute Respiratory Illness in an 11-Year-Old Boy: A Case Report.Cureus
Spatial transcriptomics maps region and isoform specific brain alterations in mdx and mdx4cv models of Duchenne muscular dystrophy.Neurobiology of disease
Trial resultA pre-post single-arm pilot trial of a digital multicomponent intervention for self-efficacy enhancement among online support groups of primary caregivers of children with neuro-muscular disorders: SEE-NMD protocol.PloS one
Willingness to adopt gene therapy for Duchenne muscular dystrophy: barriers, facilitators, and multidisciplinary readiness.Frontiers in medicine
Non-invasive adjunctive diagnostic models incorporating high-frequency ultrasound and shear wave elastography for pediatric Duchenne muscular dystrophy.Frontiers in genetics
Non-invasive assessment of microvascular changes in a Duchenne muscular dystrophy rat model with near-infrared spectroscopy.Neuromuscular disorders : NMD
ReviewEfficacy and safety of exon skipping therapies for Duchenne Muscular Dystrophy: A Systematic Review.European journal of medical genetics
A single liver-sparing myotropic AAV ameliorates muscular dystrophy in humanized mice by base editing.The Journal of clinical investigation
Does β-hydroxy-β-methylbutyrate-associated improvement in mitochondrial respiration preserve fast-twitch muscle function in Duchenne muscular dystrophy?The Journal of physiology
Exploring the relationship among nutrition, gut microbiota and musculoskeletal health in children with Duchenne muscular dystrophy: a cross-sectional study.European journal of nutrition
Case reportEarly-onset medication-related osteonecrosis of the jaw in adolescents with Duchenne muscular dystrophy receiving long-term antiresorptive combined with glucocorticoid therapy: Two case reports.Bone reports
Niche-reprogramming CAR T therapy improves skeletal muscle regeneration and function in preclinical murine models of Duchenne muscular dystrophy.Cell stem cell
Caregivers perspectives on multidisciplinary clinic visits for Duchenne and Becker muscular dystrophy.Journal of neuromuscular diseases
Early Cardiac Dysfunction in Pediatric Becker Muscular Dystrophy: A Prospective Cross-sectional Echocardiographic Study.Journal of the American Society of Echocardiography : official publication of the American Society of Echocardiography
Muscle function, linear growth, and bone health during 30 months of vamorolone, prednisone, and deflazacort treatment for Duchenne muscular dystrophy: a comparative study.Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research
Deciphering isonicotinylation and mast cell-associated pathogenic patterns in Duchenne muscular dystrophy: evidence from ensemble learning-driven multi-omics and in vitro validation.Frontiers in molecular biosciences
Phenotype rescue through the restoration of full-length dystrophin using CRISPR/Cas9 genome editing in Duchenne muscular dystrophy patient-derived iPSCs carrying the deletion of two exons.Genome medicine
Three-Year Follow-Up of Delandistrogene Moxeparvovec in Duchenne Muscular Dystrophy: Long-Term Phase 3 EMBARK Trial Results.Neurology and therapy
ReviewChronic Inflammation, Fibrotic Remodeling, and Regenerative Failure in Duchenne Muscular Dystrophy: Mechanisms and Therapeutic Opportunities.Biomedicines
Participation Patterns of Youth With Duchenne-Becker Muscular Dystrophies Across Four Settings.Canadian journal of occupational therapy. Revue canadienne d'ergotherapie
Reduced Sperm Counts in Mice with Dmd Exon 51 Frameshift Mutations.Genes
Clinical Trial Landscape of Duchenne Muscular Dystrophy in Europe: A 10-Year Retrospective Analysis.Medicina (Kaunas, Lithuania)
In vivo genome-editing therapy for Duchenne muscular dystrophy: Preclinical studies and first-in-human trial.Cell
Ventilation inhomogeneity in Duchenne muscular dystrophy before the initiation of ventilatory support: Insights from electrical impedance tomography.Pulmonology
Teaching NeuroImage: Somatic DMD Gene Mosaicism Causing Hemiatrophy in an Adult Man With Duchenne Muscular Dystrophy.Neurology
PreprintThe annexin A1 mimetic peptide Ac2-26 promotes inflammation-resolution and stimulates skeletal muscle regeneration in the mdx mouse model of Duchenne muscular dystrophy.bioRxiv : the preprint server for biology
PreprintStructural engineering of dual-targeting antisense oligonucleotides enhances exon 44 skipping for treating Duchenne muscular dystrophy.bioRxiv : the preprint server for biology
Case reportMissed Perforated Appendicitis in a Nonverbal Patient with Autism and Duchenne Muscular Dystrophy: A Case Report.Reports (MDPI)
HMB as a therapeutic strategy in Duchenne muscular dystrophy: Linking muscle function, protein homeostasis and mitochondrial bioenergetics.The Journal of physiology
Molecular Hybrids of Deflazacort and H2S Donors: From Bench to Potential Treatment for Duchenne Muscular Dystrophy.Journal of medicinal chemistry
Machine Learning Integrating Quantitative MRI and Clinical Features Predicts Loss of Ambulation in Duchenne Muscular Dystrophy.Journal of cachexia, sarcopenia and muscle
Twenty years of givinostat in Duchenne muscular dystrophy: a patient-organisation perspective.Neuromuscular disorders : NMD
Review[Duchenne muscular dystrophy-associated cardiomyopathy: advances in diagnosis and management].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics
ReviewProgress on Duchenne muscular dystrophy: from molecular mechanisms to therapeutic strategies.Yi chuan = Hereditas
ReviewBrain imaging in dystrophinopathies.Journal of neurology
From cordoned consultations to cohesive care: A service evaluation of a one-stop, multi-speciality, hospice-based Duchenne muscular dystrophy clinic, UK.Future healthcare journal
Ferroptosis-related hub genes and immune infiltration characteristics in Duchenne muscular dystrophy: a molecular framework with implications for aging-related muscle degeneration.Aging
Physiological Reference Mapping Reveals Context-Dependent Dysregulation of Regenerative Programs in Duchenne Muscular Dystrophy.Cells
Psychosocial Burden of Duchenne Muscular Dystrophy on Caregivers in Australia: A Cross-Sectional Study.Healthcare (Basel, Switzerland)
Progress in exon skipping for Duchenne muscular dystrophy.The Lancet. Neurology
Randomised trialSafety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double-blind, phase 1/2 trial.The Lancet. Neurology
In plain wordsRead moreRead the paper on PubMedReviewManaging growth and puberty concerns in individuals with Duchenne muscular dystrophy: Updated guidance and future directions.Journal of neuromuscular diseases
The Silent Window: Detecting Duchenne Cardiomyopathy Before Ejection Fraction Decline.Journal of cardiovascular pharmacology
Case reportCase Report: Duchenne muscular dystrophy-associated cardiomyopathy presenting as adolescent nonischemic heart failure-from red-flag clues to genetic confirmation and multisystem management.Frontiers in cardiovascular medicine
Altered cardiac progenitor transcriptional program accompanies NOS-associated DNA damage in a human Duchenne muscular dystrophy organoid model.Mechanisms of ageing and development
AAVcc47-μDys Gene Therapy Ameliorates Respiratory Pathology in mdx Mouse Model of Duchenne Muscular Dystrophy.The Yale journal of biology and medicine
PreprintInhibiting trained immunity in Duchenne muscular dystrophy enhances muscle regeneration and reduces fat deposition.Research square
Case reportX;7 Translocation at p21.1 and q31.31 Disrupting DMD: A Multiomics Study of a Male Dystrophinopathy Case.Human mutation
Serum albumin oxidation and inter-alpha-trypsin-inhibitor heavy chain-4 abundance correlate with the dystropathology in the rat model of Duchenne muscular dystrophy.The Biochemical journal
Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy: the NEBIDYS trial.European heart journal. Cardiovascular pharmacotherapy
Systemic responses to intravenous rAAV administration in boys with Duchenne muscular dystrophy.Frontiers in molecular medicine
ReviewFrom molecular correction to functional rescue: delivery, tissue state, and immunobiology as the principal constraints on dystrophin-restoring therapy in Duchenne muscular dystrophy.Frontiers in pharmacology
ReviewOsteoporosis in Pediatric Orthopaedics: Congenital Diseases, Medical Management, and Perioperative Considerations.Current osteoporosis reports
Answer: Acute chest pain in Duchenne muscular dystrophy.European heart journal. Acute cardiovascular care
Case reportSevere fat embolism syndrome following minor trauma in a child with Duchenne muscular dystrophy: A case report.Molecular genetics and metabolism reports
Novel Strategy for Structural Variant Genotyping by Short-Read Genomic Sequencing From Restriction-Circles: Experimental and Bioinformatics Proof-of-Concept.Human mutation
Case reportGenetic Analysis and Reproductive Intervention in a Duchenne Muscular Dystrophy Family With Cryptic Complex Chromosomal Rearrangement Identified by Structural Variation Sequencing.Human mutation
Transcriptomics-based analysis of muscle injury mechanism mediated by inflammatory imbalance in the Duchenne muscular dystrophy muscle microenvironment.Frontiers in neurology
Olesoxime Does Not Affect Mitochondrial Dysfunction and Skeletal Muscle Pathology in Dystrophin-Deficient mdx Mice.Bulletin of experimental biology and medicine
Dynamic changes in total hemoglobin concentration in the frontal region during motion-based video gameplay in patients with Duchenne muscular dystrophy: a pilot study using portable near-infrared spectroscopy (NIRS).BMC research notes
Question: Acute chest pain in Duchenne muscular dystrophy.European heart journal. Acute cardiovascular care
Dystrophin Deficiency Creates a Pro-Ferroptotic Environment in Diaphragm of mdx Mice That Is Modified by Diet and Glucocorticoid Treatment.Biomolecules
Cross-Species Transcriptomic Integration Identifies a Conserved Molecular Core and Regulatory ceRNA Network in Duchenne Muscular Dystrophy.Genes
Gene-Corrected Human iPSC-Derived Cardiomyocytes and Skeletal Muscles Reveal Partial Dystrophin Dp427 Preservation and Cardiac Dp116 Expression in Duchenne Muscular Dystrophy Patient With a Splice-Site Mutation in Intron 68 (c.9975-1G>T).Circulation. Genomic and precision medicine
ReviewA Multidisciplinary Approach to the Anesthetic Management of Patients With Duchenne Muscular Dystrophy.Pediatric pulmonology
Exploring Awareness of and Self-Reported Adherence to Neuromuscular Clinical Practice Guidelines Among Australian and New Zealand Health Professionals: A Cross-Sectional Survey Study.Journal of evaluation in clinical practice
ReviewA structured approach to address variability in the global management of adrenal insufficiency in Duchenne muscular dystrophy: the 2025 PJ Nicholoff steroid protocol.Frontiers in neurology
PreprintRNA trans-splicing treatment for Duchenne muscular dystrophy.bioRxiv : the preprint server for biology
Case reportCongenital Clubfoot as an Early Manifestation of Duchenne Muscular Dystrophy?Muscles (Basel, Switzerland)
ReviewCharacterization of Dystrophin-Related Syndromes: Carriers, DMD, and BMD.Muscles (Basel, Switzerland)
Sleep Quality among Family Caregivers of Children with Duchenne Muscular Dystrophy.Neuropediatrics
ReviewAdrenal insufficiency in individuals with Duchenne muscular dystrophy treated with glucocorticoids: Insights from the past, current challenges, and future directions.Journal of neuromuscular diseases
Assessment of muscle damage in patients with advanced Duchenne muscular dystrophy based on serum creatinine and the height-to-creatinine ratio.Frontiers in neurology
Mutational Spectrum of DMD Gene Variants and Cascade Screening Outcomes in the Central-Eastern Black Sea Region of Türkiye: A Single-Centre Retrospective Study.Molecular biology reports
Long-term imoxin treatment does not attenuate disease severity in dystrophic diaphragm of male mice.Physiological reports
The impact of an electric hand warmer on powered wheelchair mobility in men with duchenne Muscular dystrophy.Disability and rehabilitation. Assistive technology
Leveraging external control for accelerating development: A case study from cell gene therapy in Duchenne muscular dystrophy.Journal of biopharmaceutical statistics
"Improving the beneficial effects of HDAC8 inhibition by simultaneously activation of SIRT1 in a zebrafish model of Duchenne muscular dystrophy.Pharmacological research
ReviewThe role of the respiratory physiotherapist in the management of Duchenne muscular dystrophy.Breathe (Sheffield, England)
Spectrum and carrier frequency of DMD in Yueyang, China: a population-based analysis using NGS and MLPA.Frontiers in genetics
Sociodemographic and Clinical Profile of Adult Males With Duchenne Muscular Dystrophy.Muscle & nerve
Development of an AAV-Encoded Adenine Base Editor for Duchenne Muscular Dystrophy.Human gene therapy
Mental health support for informal carers of people living with Duchenne muscular dystrophy: Lived experience perspectives.Patient education and counseling
Safety and efficacy of AAV-based mini- and micro-dystrophin gene therapies in Duchenne muscular dystrophy: a systematic review and meta-analysis of clinical trials.Journal of medical genetics
ReviewMagnetic Mitohormesis as a Potential Non-Invasive Restorative Therapy for X-Linked Muscular Dystrophies.International journal of molecular sciences
Novel Therapeutic Frontiers in Duchenne Muscular Dystrophy: Gene Therapy, Exon Skipping, and Stem Cell Approaches.Current pharmaceutical design
Should taurine be pursued further as a supportive nutraceutical for Duchenne muscular dystrophy? Gathering evidence in a zebrafish disease model to confirm or refute the benefits - Stage 1 Report.Frontiers in nutrition
Risk factors for abnormal bone mineral density in Duchenne muscular dystrophy and the association of regular rehabilitation with bone health.Frontiers in pediatrics
Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy.Molecular therapy : the journal of the American Society of Gene Therapy
Positive allosteric modulator of SERCA pump NDC-1171 attenuates cardiac functional decline in mouse model of Duchenne muscular dystrophy.Function (Oxford, England)
Respiratory Care Trajectory in Patients With Duchenne Muscular Dystrophy in the Advanced Stage: A Retrospective Single-Center Study.Muscle & nerve
Functional assessments used in routine care for duchenne muscular dystrophy in the United States: A survey of certified duchenne care centers.Journal of neuromuscular diseases
Engineering suppressor tRNAs for effective treatment of Duchenne muscular dystrophy.Science advances
Integrative multi-omics analysis reveals lipid/metabolite dysregulation and temporal decoupling in disease progression.Scientific reports