Wearable technologies in clinical trials for drug development: trends and emerging opportunities.

Correlative multimodal imaging for microscale spatial mapping of collagen-gene activity interactions in human tissues.

Unmet Needs in the Care of Patients with Duchenne Muscular Dystrophy in Brazil.

Optical Genome Mapping and Long-Read Sequencing Identifies a Novel Dystrophin Gene Inversion in a Patient With Duchenne Muscular Dystrophy.

Global MyoG research 2004-2024: a bibliometric analysis of trends and translational implications.

Investigation of Walking Ability in 161 Patients With Duchenne Muscular Dystrophy.

Thorough QT Study on the Effect of Therapeutic and Supratherapeutic Dosing of Givinostat in Healthy Volunteers.

Motor Function and Growth Outcomes With Early Corticosteroid Initiation in Duchenne Muscular Dystrophy: An Adjusted Cross-Trial Comparison.

Viscoelastic properties of dystrophin-deficient mouse skeletal muscles are resilient to isometric fatiguing exercise.

Euglycemic ketoacidosis in a nondiabetic patient with Duchenne muscular dystrophy on dapagliflozin: comment.

CTLA4-Ig reduces proliferation and inflammatory gene expression in muscle fibroblasts, corresponding to less fibrosis and inflammation in mdx muscular dystrophy.

Clinico-genetic heterogeneity in Pakistani families affected with muscular dystrophies.

Sirolimus for the treatment of steroid-refractory hepatotoxicity following AAV gene therapy in patients with Duchenne muscular dystrophy.

Co-occurring DMD, GJA1, and novel FYCO1 variants in a proband from a consanguineous oculodentodigital dysplasia family: a rare multi-locus case report.

Impact of C4BPA on Muscle progenitor cell differentiation: insights for Duchenne muscular dystrophy treatment.

Myofibre Density Reveals a Critical Threshold Around Age 6 in Steroid-Naïve Duchenne Muscular Dystrophy: A Retrospective Observational Study.

CRISPR Gene Editing for Nucleotide Repeat Expansion Disorders: A Systematic Review of Preclinical and Clinical Evidence.

Rethinking Corticosteroid Therapy in Pediatric Neurology.

Economic burden of Duchenne muscular dystrophy from a societal perspective in Mumbai, India.

Exosome-Mediated Benefits of Cell Therapy in Mouse and Human Models of Duchenne Muscular Dystrophy.

Burden of illness of Duchenne muscular dystrophy in Belgium: A retrospective, descriptive, cross-sectional study.

Skipping the Biopsy: Real-World Experience of Whole-Exome Sequencing as First-Tier Testing in Pediatric Muscular Disorders.

Mutation of leucine 170 alters the subcellular distribution, neurite outgrowth and three-dimensional structure of dystrophins Dp71 and Dp40.

PTBP1 inhibition reprograms myogenesis to rescue impaired muscle regeneration in mdx mice through correcting E2A splicing.

Dystrophin-Deficient Cardiomyopathy Due to a Novel Hemizygous DMD Indel Variant.

Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study.

An Assessment of Paediatricians' Knowledge and Perspectives of Duchenne Muscular Dystrophy in Oman.

Duchenne Muscular Dystrophy Presenting as Acute Flaccid Paralysis: Authors' Reply.

Bioengineered AAV9 and Optimised Microdystrophin Vectors Augment Phenotypic Rescue in a Murine Model of Duchenne Muscular Dystrophy.

Evaluation and management of DMD gene copy number variations detected by prenatal SNP-array testing.

Real-world walking speed as a digital biomarker and outcome measure for clinical trials-a systematic review, regulatory status and future directions.

Status of Clinical Care of Duchenne Muscular Dystrophy: Global Perspective and Situation in India.

Longitudinal ankle range of motion and functional decline in Duchenne muscular dystrophy.

Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy.

Fat embolism syndrome in Duchenne muscular dystrophy: an underdiagnosed complication.

Randomised trial of Aureobasidium pullulans-produced beta 1,3-1,6-glucans in patients with Duchenne muscular dystrophy: favourable changes in gut microbiota and clinical outcomes indicating their potential in epigenetic manipulation.

Molecular Insights and Orthopedic Management in Muscular Dystrophies: A Comprehensive Review.

Potential Involvement of Ferroptosis in Duchenne Muscular Dystrophy-Associated Cardiomyopathy.

Effects of Bisphosphonates on Bone Micro-Architecture of Children With Duchenne Muscular Dystrophy: A Prospective Comparative Study.

Pan-immune-inflammation value as a predictor of loss of ambulation in duchenne muscular dystrophy: a retrospective cohort study.

Management of Duchenne Muscular Dystrophy in Clinical Practice: A Survey-Based Study in Spain.

Analysis of adverse event reporting with casimersen: a pharmacovigilance study based on the United States food and drug administration adverse event reporting system database.

Functional and structural pathologies in skeletal muscle of a rat model of Duchenne muscular dystrophy.

Machine learning for site risk prediction in clinical trials: development, external validation, and operational application in site qualification.

Duchenne Muscular Dystrophy Presenting as Acute Flaccid Paralysis: Correspondence.

Population-Based Investigation of DMD Genotype and Neurodevelopmental Concerns in Duchenne Muscular Dystrophy.

Evaluation of Readthrough Efficiency of Negamycin Derivatives against Nonsense Mutations in Muscular Dystrophy Genes.

The Paradox of Life Extension with Traditional Cost-Effectiveness Analysis: A Case Study with Duchenne Muscular Dystrophy.

Senescent-Like Myofibers Contribute to Anti-Regenerative Cytokine Signaling in Duchenne Muscular Dystrophy.

Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study.

Early Cardiac Manifestations as the Initial Presentation of Duchenne Muscular Dystrophy in Infancy.

Trunk control status in children with neuromuscular disorders and typically developing children: Is there a measurable difference?

A simple and highly sensitive LC-MS/MS bioanalytical method for phosphorodiamidate morpholino oligonucleotides in plasma.

The Impact of Trunk Control and Balance on Functional Skills in Ambulatory Children With Duchenne Muscular Dystrophy.

Test the grandfather! Incidental in-frame DMD deletions in three asymptomatic families.

Safety and Efficacy of Tamoxifen in Patients with Duchenne muscular dystrophy: open Label Extension of TAMDMD Trial.

Motor Function Changes in Duchenne Muscular Dystrophy: A Case Series Using Conventional and Spinal Muscular Atrophy-Based Assessments During Viltolarsen Treatment.

Clinical diagnosis and genetic analysis of a rare case of Duchenne muscular dystrophy and spinal muscular atrophy.

MLDP-AS: an optimized next-generation sequencing assay for enhanced detection of technically challenging variants in expanded carrier screening.

A Narrative Review of Necroptosis in Neuromuscular Junction Disorders: Pathogenesis and Therapeutic Strategies.

Uncommon Associations in Duchenne Muscular Dystrophy: Coexistence of Patent Foramen Ovale and Ocular Anomalies.

A Translational Roadmap for Neurological Nonsense Mutation Disorders.

Macrophage Infiltration, Activation, and Therapeutic Implication in Skeletal Muscle Injury and Repair.

Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management.

Patient reported outcome measures in spinal muscular atrophy and duchenne muscular dystrophy: review of instruments and their inclusion in clinical and regulatory processes.

Prognostic value of right ventricular-pulmonary artery coupling in patients with muscular dystrophies.

Diagnostic Cut-off Values for Newly Developed Noninvasive Elastance-Based Indices in Duchenne Muscular Dystrophy-Associated Cardiomyopathy.

Patient-Derived 3D Bioprinted Cardiac Organoid Constructs Reveal Key Pathological Features of Duchenne Muscular Dystrophy.

ALT/CK Ratio as an Early Marker of Liver Injury After Gene Therapy in Duchenne Muscular Dystrophy.

Lumbopelvic stabilization-based physiotherapy and rehabilitation and urotherapy for lower urinary tract dysfunction in Duchenne Muscular Dystrophy: a randomized controlled trial.

Early Diagnosis of Duchenne Muscular Dystrophy Requires Newborn Screening for CK, and in the Event of Paresis, Relevant Investigations.

Evaluation of protein expression and oxidative stress index in Duchenne muscular dystrophy.

Screening for brain-related comorbidities in Duchenne muscular dystrophy: Construction, reliability, and validity of the BIND screener.

Implementation of Newborn Screening for Duchenne Muscular Dystrophy.

Direct AMPK Activation Confers Mutation-Independent Therapeutic Benefit in Duchenne Muscular Dystrophy.

Potential benefits of Ayurveda in Duchenne muscular Dystrophy: A case based analysis.

Neuromuscular and neuromechanical assessments of respiratory performance in the mdx mouse model of Duchenne muscular dystrophy across the natural history of disease.

Symptomatic Pneumoperitoneum After Percutaneous Radiological Gastrostomy in Patient With Duchenne Disease Dependent on Non-Invasive Mechanical Ventilation.

Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy.

Identification and functional validation of intracellular protein partners of phosphorothioate splice-switching oligonucleotides using AP-MS.

Rare DMD Gene Duplication in a Lebanese Child With Duchene Muscular Dystrophy.

From lipid switch to tissue repair: how resolvins reprogram macrophage polarization and function.

Evaluation of repolarization abnormalities with 12-lead ECG and 24-hour Holter ECG monitoring in patients with Duchenne muscular dystrophy.

The Promise and Pitfalls of AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy.

Making brain health routine in Duchenne muscular dystrophy: What the BIND screener adds-and what it is not designed to do.

Psychological and functional predictors of chronic pain outcomes in youth with Duchenne muscular dystrophy.

Reframing fibrosis as a barrier to regeneration and gene delivery in muscular diseases.

Myostatin inhibition with orally administered Lactobacillus casei expressing a modified human myostatin protein: functional benefits and translational potential in advanced Duchenne muscular dystrophy.

Impaired stem cell migration and divisions in Duchenne muscular dystrophy revealed by live imaging.

Twisting Paths: The Paradox of Fiber Branching in Muscle Regeneration.

Inhibition of TRPC3-Nox2 Complex Formation Ameliorates Skeletal Muscle Atrophy.

State-of-the-Art Research and New Pharmacological Perspectives on Renal Involvement in Duchenne Muscular Dystrophy: A Narrative Review.

Statistical Genetics of DMD Gene Mutations in a Kazakhstan Cohort: MLPA/NGS Variant Validation and Genotype-Phenotype Modelling.

Brain Matters in Duchenne Muscular Dystrophy: DMD Mutation Sites and Their Association with Neurological Comorbidities Through Isoform Impairment.

Beginning of a new era of synthetic messenger RNA therapeutics: Comprehensive insights on mRNA drug design, development and applications.

Systemic distribution of tricyclo-DNA antisense oligonucleotide following intratracheal instillation in the mouse.

A Rare Co-occurrence of Duchenne Muscular Dystrophy and Glycerol Kinase Deficiency Associated With Xp21 Contiguous Gene Deletion Syndrome: A Case Report.

Quantitative MRI Findings and Their Relationship to Muscle Histopathology and Ambulatory Clinical Function in Duchenne Muscular Dystrophy.

Meeting report: Expanding access to advanced cardiac therapies, including ventricular assist devices (VADs) and heart transplantation in muscular dystrophy.

Can Real-Time Three-Dimensional (Four-Dimensional) and Speckle-Tracking Echocardiography Predict Cardiac Involvement Detected by Magnetic Resonance Imaging in Children with Duchenne Muscular Dystrophy?

Advancing cardiac monitoring in adult Duchenne muscular dystrophy: longitudinal insights and real-world challenges.

Incremental Disease Burden (Healthcare Costs and Resources) of Duchenne Muscular Dystrophy in the US: A Matched Cohort Analysis.

Engineering the MmeFz2-ωRNA system for efficient genome editing through an integrated computational-experimental framework.

Systematic analysis of the adverse effects of used clinical antisense oligonucleotide drugs in DMD patients based on the FAERS database.

PPMO-based exon skipping therapy improves respiratory function in the mdx mouse model of Duchenne muscular dystrophy.

Humanin improves bone health in a glucocorticoid-treated mouse model of Duchenne muscular dystrophy.

Role of Cardiovascular Magnetic Resonance in Diagnosis and Management of Muscular Dystrophies.

Parkin overexpression attenuates muscle atrophy and improves mitochondrial bioenergetics but not histological features of Duchenne muscular dystrophy in mice.

Regenerative Index reveals declining muscle regeneration in paediatric patients with Duchenne muscular dystrophy.

Assessing Delays in Time to Diagnosis of Duchenne Muscular Dystrophy: A Survey of Current Primary Care Practices.

Obestatin Treatment Counteracts Muscle Wasting by Reactivation of Autophagy in Duchenne Muscular Dystrophy.

[Expert consensus on respiratory rehabilitation strategies for Duchenne muscular dystrophy (2026)].

Multiple modes of AFM reveal distinct mechanical properties for dystrophin and utrophin not manifest by small fragments.

Muscle meets Lysosomes: emerging strategies in muscular dystrophy.

Emerging therapeutic strategies in muscular dystrophy: an updated review on pathogenesis and treatment advances.

Genotype-Dependent Morpho-Mechanical Profiling of Patient-Derived Human Myotubes on Nanogrooved Substrates.

Bladder dysfunction in Duchenne muscular dystrophy: A narrative review.

Hyperactivity, compulsive-like behaviours, and impaired flexibility in mouse models of Duchenne muscular dystrophy.

Environmental Determinants of Participation in Children With Duchenne Muscular Dystrophy.

Caregiver burden in Duchenne muscular dystrophy in Europe, Japan, and the United States - a real-world study.

Two-Year Outcomes Following Delandistrogene Moxeparvovec Treatment in Ambulatory Patients with Duchenne Muscular Dystrophy: Phase 3 EMBARK Trial.

Spectrum of Osteoporosis Etiologies with Associated Vertebral Compression Fractures in Children: Analysis of 11 Cases.

Investigating the role of EGFR signalling in muscle dystrophies: implications for Duchenne muscular dystrophy.

RNA-Based Therapies for Treating Monogenic Cardiomyopathies.

cPLA2 in musculoskeletal and autoimmune diseases: Molecular mechanisms and therapeutic insights.

Muscle involvement in women carrying pathogenic DMD gene variants: A 6.5-year follow-up study.

Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone.

Unravelling the Complications of Dilated Cardiomyopathy in Duchenne Muscular Dystrophy: From Molecular Pathways to Disease Management.

Cardiac and skeletal muscle delivery of biotherapeutics with a blood vessel epicardial substance-targeting peptide.

Evaluation of a serum protein signature as monitoring biomarker for Duchenne Muscular Dystrophy in a long-term clinical trial with corticosteroids.

A new dystrophin-deficient rat model mirroring exon skipping in patients with DMD exon 45 deletions.

Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.

Variant burden and severity of cardiomyopathy in patients with DMD-related Duchenne muscular dystrophy.

Interpreting Neonatal hyperCKemia Identified Through Duchenne Muscular Dystrophy Newborn Screening: A Predictive Model Based on Maternal, Labor, Delivery and Newborn Factors.

Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling.

Mineralocorticoid receptor antagonists reduce inflammatory signaling independent of myofiber mineralocorticoid receptor.

Knock-out of specific DMD gene isoforms in the parental hESC line SA001 using CRISPR/Cas9.

Bridging the gap in sexuality and neuromuscular disorders: a scoping review of an overlooked but crucial topic.

Inflammation in a severe model of dystrophic cardiomyopathy contains a high proportion of T cells that contribute to onset of pathology.

Proximity to Healthcare Services Among Individuals With Duchenne Muscular Dystrophy.

mGem: AAV, from almost a virus to an awesome vector-or is it?

CTRP1 regulates skeletal muscle differentiation through quality control of mitochondrial dynamics and function.

Disease exacerbation in human DMD MYOrganoids enables gene therapy evaluation and unveils persistence of fibrotic activity.

The complexity of dystrophin transcription and processing: implications of transcript imbalance on dystrophin gene targeting strategies.

Inhibiting Myozenin 1 Attenuated Muscular Dystrophy Pathology in mdx Mice by Enhancing Calcineurin Activity.

Targeting autophagy in Duchenne muscular dystrophy: mechanistic insights and emerging therapeutic strategies.

Inhibition of HuR/ELAVL-1 attenuates fibrotic progression in Mdx mice with dilated cardiomyopathy.

Behavioral improvement in dystrophic mdx23 mouse following repeated antisense oligonucleotides injections.

Fishing for novel HDAC inhibitor compounds to treat Duchenne muscular dystrophy.

Evaluation of a control paradigm allowing heart rate guided rehabilitative exercise for boys with Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy in the Republic of North Ossetia-Alania: Epidemiological Study, Diagnostic Issues, and Treatment Prospects.

The Balancing Act of Paid Work and Caregiving in Duchenne Muscular Dystrophy (DMD): Results from a Cross-sectional Survey.

[Application of multi-technique in combined for the detection and prenatal diagnosis of families affected with Duchenne muscular dystrophy].

Age-related differences in hot and cold executive functions in boys with Duchenne muscular dystrophy: longitudinal individual changes and age-group comparisons across childhood and adolescence.

Transgene-induced cardiotoxicity in high-dose AAV gene transfer.

Becker muscular dystrophy (BMD) is caused by a dystrophin missense mutation in the original family of Becker and Kiener.

β-Hydroxy-β-methylbutyrate enhances fast-twitch muscle and mitochondrial function, histopathology and mTORC1 signalling in the mdx dystrophic mouse.

Diagnostic Precision in Pediatric Neuromuscular Disorders: A Case Study of Bethlem Myopathy Mimicking Duchenne Muscular Dystrophy.

Acute Adverse Events Following Intravenous Bisphosphonate Infusion Are Uncommon in Patients With Duchenne Muscular Dystrophy Previously Treated With Oral Bisphosphonates.

Identification of myokines associated with the pathological stress response in the mdx mouse model of Duchenne muscular dystrophy.

Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice.

Spatially Resolved Profiling of Compartmentalized Muscle and Brain Inflammation.

Morpholino-RNA duplex exhibits robust, sustained, and safe steric-block antisense activity by intracerebroventricular and intrathecal injection.

Endocrine and metabolic complications in a national cohort of Slovene children and adolescents with Duchenne muscular dystrophy: real-world criteria for transition to vamorolone therapy.

Quantitative Magnetic Resonance Imaging of Gluteal Muscle Groups Detects Stage-Specific Progression and Early-Stage Damage in Duchenne Muscular Dystrophy: A 12-Month Longitudinal Study.

Heart Transplantation and Ventricular Assist Device in Duchenne Muscular Dystrophy: A New Era.

SRSF2 is upregulated in Duchenne muscular dystrophy and impairs myoblast autophagy by alternatively splicing HUWE1.

Muscle satellite cell editing by LNP-CRISPR-Cas9 to resist muscle injury.

Long-Term Safety and Efficacy of Systemic DT-DEC01 Cell Therapy in Non-Ambulatory Duchenne Muscular Dystrophy Patients: a 24-Month Clinical Evaluation.

Development of a cost-effectiveness analysis model for Duchenne muscular dystrophy utilizing the national registry in Japan.

Validation of an in vitro muscle platform to evaluate myogenesis and calcium handling in control and dystrophic human myotubes.

HDAC11 deficiency improves muscle phenotype in a Duchenne muscular dystrophy murine model by reducing inflammation and fibrosis.

Duchenne Muscular Dystrophy: Clinical Innovations, Ethical Considerations, and Evolving the Path to Adulthood.

Delandistrogene Moxeparvovec gene therapy for Duchenne muscular dystrophy: the way forward in South Asian countries.

Six-Minute Activity-95th Centile, a Novel Wearable-Derived Clinical Outcome Assessment for Duchenne Muscular Dystrophy.

Acute Gastric Dilatation in a 16-Year-Old Patient With Duchenne Muscular Dystrophy: A Case Report and an Updated Literature Review.

Genetic counseling, prenatal diagnosis and newborn screening in Duchenne muscular dystrophy.

Other innovative therapies in Duchenne muscular dystrophy.

Gene therapy in Duchenne muscular dystrophy.

Corticosteroid treatment in Duchenne muscular dystrophy.

Neuropsychological management in Duchenne muscular dystrophy: A critical overview and future directions.

Orthopedic management in Duchenne muscular dystrophy.

Genetics and pathophysiology of Duchenne muscular dystrophy.

Respiratory care in Duchenne muscular dystrophy.

Cardiac care in Duchenne muscular dystrophy.

Multidisciplinary management of Duchenne muscular dystrophy from childhood to adulthood.

Duchenne muscular dystrophy: the French Dystrophinopathies Registry (DYS Registry).

Duchenne muscular dystrophy in 2025.

Targeting Skeletal Muscle in Duchenne Muscular Dystrophy: Integrating in Silico and Experimental Approaches to Sodium-Glucose Cotransporter-2 Inhibition.

Antisense and gene therapy trials in Duchenne muscular dystrophy.

Upper limb function and quality of life in Duchenne muscular dystrophy: a cross-sectional study in Chile.

Tonic GABAA receptor currents in Cerebellar Purkinje cells of wild-type and DMDmdx mice.

Transcytotic transportation of size-controlled nanocarriers into dystrophic skeletal muscle leads to therapeutic outcome in mice.

U.S. health plan coverage of Neuromuscular Disease Therapies: An assessment of policy availability and restrictions.

Targeting lysosomal damage in Duchenne muscular dystrophy.

[Duchenne de Boulogne: Pioneer of Neurology].

Optical genome mapping identifies a balanced inversion disrupting DMD in a patient with Duchenne muscular dystrophy.