What researchers published on Duchenne muscular dystrophy
737 papers, newest first.
Showing 180 of 737
The longer, the better? Optimizing dystrophin constructs for cardiac gene therapy in Duchenne muscular dystrophy.Molecular therapy. Advances
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
ReviewDuchenne muscular dystrophy: from gene to gene-ius therapies.Skeletal muscle
MicroRNA-mRNA Networks in Skeletal Muscle of Tailored Pig Models for Dystrophinopathies.Journal of cachexia, sarcopenia and muscle
Beyond the Diagnosis: A Comprehensive Case Report on Duchenne Muscular Dystrophy.Case reports in critical care
A Registry-Based Perspective of Interventional Clinical Trials for Duchenne Muscular Dystrophy.Cureus
Histological, functional, and behavioral comparison of three Duchenne muscular dystrophy mouse models.Neuromuscular disorders : NMD
Case reportA 6-Year-Old With Becker Muscular Dystrophy and Catatonia.Pediatrics
Meta-analysisImproving survival in Duchenne muscular dystrophy across eras: a systematic review and cumulative meta-analysis.Journal of neurology
In plain wordsRead moreRead the paper on PubMedCase reportDelayed diagnosis and genetic testing in spinal muscular atrophy: a case series.Annals of medicine
Muting lateralized Cx43 hemichannels to rescue the stressed Duchenne heart.Cardiovascular research
Case reportVolatile-Free Total Intravenous Anesthesia for Major Gynecologic Surgery in a Manifesting Female Carrier of Becker Muscular Dystrophy: A Case Report.Cureus
Maternal rumination and well-being in Duchenne muscular dystrophy: Links to children's function and balance.Journal of pediatric nursing
Protein intake is associated with adiposity and quality of life in adults with Becker muscular dystrophy.Clinical nutrition ESPEN
ReviewEvidence Regarding Duchenne Muscular Dystrophy Newborn Screening.Pediatrics
A Nomogram Integrating Clinical and Cardiac Imaging for Predicting Short-Term Left Ventricular Ejection Fraction Decline in Patients with Duchenne Muscular Dystrophy.International journal of general medicine
Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to DMD exon 16 skipping.Molecular therapy. Nucleic acids
Givinostat enhances antisense oligonucleotide efficacy in the mdx52 model of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids
Exon-skipping therapies for DMD in Kazakhstan: Progress and challenges.Journal of neuromuscular diseases
Case reportFunctional Trajectory and Quality of Life Divergence Following Surgical Versus Conservative Management of Achilles Tendon Contracture in Monozygotic Twins with Duchenne Muscular Dystrophy: A Case Report.Children (Basel, Switzerland)
Neuropsychological and Emotional-Behavioral Profiles in Pediatric Duchenne Muscular Dystrophy: A Single-Center Clinical Study.Children (Basel, Switzerland)
Influence of community disadvantage on cognitive performance in son-mother dyads in Duchenne muscular dystrophy.Brain communications
Age-Dependent Effect of Myostatin Blockade in the mdx Mouse Model of Duchenne Muscular Dystrophy (DMD).Muscles (Basel, Switzerland)
Sauchinone-loaded chitosan nanoparticles simultaneously suppress NF-κB-driven inflammation, activate the Nrf2/HO-1 axis, and restore sarcolemmal utrophin in a murine model of Duchenne muscular dystrophy.Colloids and surfaces. B, Biointerfaces
Balancing Benefits and Risks: Long-term Use of Daily versus Intermittent Corticosteroids in Duchenne Muscular Dystrophy.Journal of neurology, neurosurgery, and psychiatry
Derivation of a new induced pluripotent stem cell line ORIONi004-A for modelling Duchenne muscular dystrophy.Stem cell research
Bridging Duchenne muscular dystrophy therapy from rhesus monkeys to patients.Clinical and translational medicine
Four new mouse models of Duchenne muscular dystrophy with clinically relevant exon deletions in the human DMD gene.Disease models & mechanisms
PreprintAAV and lentiviral transduction in Duchenne muscular dystrophy cardiomyocytes activate cell stress responses.bioRxiv : the preprint server for biology
Beyond dystrophin: cell therapy for Duchenne muscular dystrophy.Lancet (London, England)
LKB1 Dysregulation in Duchenne Muscular Dystrophy Models: Disease Specificity and Epigenetic Control by HDAC Inhibitors.Annals of the New York Academy of Sciences
Integrating Genetic Modifier Genotype With Serum Proteomics in Duchenne Muscular Dystrophy Clinical Trials Links LTBP4 Genetic Modifier to IL-23/CD93 Pathways in Muscle.American journal of medical genetics. Part A
Erratum: Microdystrophins partially rescue deficits of Duchenne muscular dystrophy iPSC-cardiomyocytes.Molecular therapy. Advances
Lipin1 restores nNOS sarcolemmal localization and improves fatigue resistance in Duchenne muscular dystrophy.American journal of physiology. Cell physiology
ATP6AP2 dysregulation as a central hub in DMD pathogenesis: An integrated multi-omics and therapeutic study.International immunopharmacology
Generation and characterization of three human induced pluripotent stem cell lines from patients with Duchenne muscular dystrophy.Stem cell research
Prime Editing Mediated Generation and Correction of the mdx5cv Mutation Restores Dystrophin Expression in Myoblasts.International journal of molecular sciences
Cannabidiol-induced Heme oxygenase-1 contributes to modulate the phenotype of hiPSC-derived cardiac fibroblasts from patients with Duchenne muscular dystrophy.Redox biology
An antisense method for efficient exon skipping and its application to Duchenne muscular dystrophy.Proceedings of the National Academy of Sciences of the United States of America
Case reportFat Embolism Syndrome Following Elective Orthopedic Surgery in a Patient With Duchenne Muscular Dystrophy.Cureus
Reduced Cx43 S-nitrosylation protects against cardiac stress-induced arrhythmias and myocardial injury in Duchenne muscular dystrophy.Cardiovascular research
Meta-analysisPrevalence and risk factors of bone fragility in duchenne muscular dystrophy: A systematic review and meta-analysis.Osteoporosis international : a journal established as result of cooperation between the European Foundation for Osteoporosis and the National Osteoporosis Foundation of the USA
In plain wordsRead moreRead the paper on PubMedClinical Utility and Measurement Properties of Balance Measures in Duchenne Muscular Dystrophy: A Scoping Review.Pediatric neurology
Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy.Molecular therapy. Nucleic acids
Evaluation of antibody reactivity to dystrophin products: does Dp40 really exist?Neuromuscular disorders : NMD
ReviewYoga as an Adjunctive Intervention for Duchenne Muscular Dystrophy: A Scoping Review.International journal of yoga
Psychosocial Care Utilization Among Patients With Duchenne Muscular Dystrophy.Pediatric neurology
Randomised trialEffects of Virtual Reality Versus Whole-Body Vibration on Muscle Strength and Balance in Children with Duchenne Muscular Dystrophy: a Randomized Controlled Trial.NeuroRehabilitation
In plain wordsRead moreRead the paper on PubMedRandomised trialPrednisone, not vamorolone, suppresses novel serum bone and cartilage biomarkers associated with growth failure in children with Duchenne muscular dystrophy.Scientific reports
In plain wordsRead moreRead the paper on PubMedSwallowing Dysfunction and Histopathological Changes in a Rat Model of Duchenne Muscular Dystrophy.Dysphagia
ReviewEchocardiography in duchenne muscular dystrophy: a call for consistency and standardisation of reporting.Echo research and practice
Evaluating exon skipping in the central nervous system in Duchenne muscular dystrophy using spatial transcriptomics.iScience
Quality Improvement to Promote Advance Care Planning for Young Adults with Duchenne Muscular Dystrophy.Palliative medicine reports
Improving Nocturnal Non-Invasive Positive Pressure Support Adherence in Children and Young Adults With Duchenne Muscular Dystrophy.Pediatric pulmonology
Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial.Neurology
ReviewCytoskeleton-Membrane Uncoupling in Duchenne Muscular Dystrophy: Implications for Newborn Screening and Early Protection.Cytoskeleton (Hoboken, N.J.)
Gamified occupational therapy program for adolescents with Duchenne muscular dystrophy: A study protocol for randomized controlled trial.Assistive technology : the official journal of RESNA
Case reportInpatient rehabilitation admission for a patient with Duchenne muscular dystrophy status post Achilles tendon release.PM & R : the journal of injury, function, and rehabilitation
Observational studyCharacterizing obsessive-compulsive features in Duchenne muscular dystrophy: a DSM-5 approach.Neuromuscular disorders : NMD
Case reportRole of Finerenone in Duchenne Muscular Dystrophy in a 45-Year-Old Man: Outcome and Brief Review.JACC. Case reports
Randomised trialDeramiocel heart-derived cellular therapy in advanced Duchenne muscular dystrophy (HOPE-3): a phase 3, randomised, double-blind, placebo-controlled trial.Lancet (London, England)
In plain wordsRead moreRead the paper on PubMedThe Subclinical Window for Cardioprotection in Duchenne Muscular Dystrophy: A Systematic Review and Meta-analysis.Journal of cardiovascular pharmacology
Duchenne Muscular Dystrophy Under Three Years of Age.Children (Basel, Switzerland)
Niclosamide reprograms macrophages and improves muscle integrity in Duchenne muscular dystrophy models.Biochemical pharmacology
Recalibrating Therapeutic Priorities for Duchenne Muscular Dystrophy: A Critical Synthesis of Approved and Emerging Strategies Through the Lens of an Underrepresented Population.Genes
Engineered exosomes improve myocardial cell membrane integrity and heart function in dystrophic cardiomyopathy.Clinical and translational medicine
Case reportDystrophin-Deficient Muscular Dystrophy in a Jack Russell Terrier With a Large Deletion in the Canine DMD Gene.Veterinary medicine and science
Evaluation of Trendelenburg and Duchenne signs by experimentally induced gluteal muscle paralysis.Clinical biomechanics (Bristol, Avon)
Effects of intermittent corticosteroids on scoliosis, bone density, and vertebral fractures in duchenne muscular dystrophy.Journal of neuromuscular diseases
Microdystrophins partially rescue deficits of Duchenne muscular dystrophy iPSC-cardiomyocytes.Molecular therapy. Advances
GDF5 modulation of MuSC pool as a potential therapeutic benefit for DMD.Molecular therapy. Nucleic acids
Case reportCase Report: The effect of early initiation of eteplirsen treatment on the cardiac and motor disease course in an individual with Duchenne muscular dystrophy.Frontiers in pediatrics
Meta-analysisAccuracy of the molecular diagnosis of duchenne and becker muscular dystrophy: A systematic review with meta-analysis.PloS one
In plain wordsRead moreRead the paper on PubMedBeyond Teleconsultation: Exploring the Role of Mobile Health Technologies in Duchenne Muscular Dystrophy.JMIR formative research
The Impact of Clinical Trial Participation on Quality of Life in Duchenne Muscular Dystrophy.Pharmaceutical medicine
Clinical spectrum and ascertainment pathways in pediatric female dystrophinopathy.Italian journal of pediatrics
Prevalence of Depression among Children with Duchenne Muscular Dystrophy: A Cross-sectional Survey Study.Annals of Indian Academy of Neurology
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.Neurobiology of disease
AI-powered therapeutic aptamer drug discovery: Targeting the CT-domain of CTGF for duchenne muscular dystrophy.Acta pharmaceutica Sinica. B
Randomised trialEvaluation of a serum protein signature as monitoring biomarker for Duchenne muscular dystrophy in a long-term clinical trial with corticosteroids.Skeletal muscle
In plain wordsRead moreRead the paper on PubMedClinical, pathological, and genetic characteristics of 23 DMD patients in northern China.Orphanet journal of rare diseases