duchenne-muscular-dystrophy

Documents about duchenne-muscular-dystrophy

• Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)#828639ClinicalTrials.gov
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)#862999ClinicalTrials.gov
• Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children With DMD/SMA#837754ClinicalTrials.gov
• EMILIN1 emerges as a TGFβ/SETDB1-regulated secreted biomarker in Duchenne muscular dystrophy#273947HAL (France)
• Safety and Efficacy of P-188 NF in DMD Patients#813407ClinicalTrials.gov
• Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study#1010655ClinicalTrials.gov
• Optimum Insufflation Capacity in NMD#860868ClinicalTrials.gov
• A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74#870870ClinicalTrials.gov
• Yoga as an Adjunctive Intervention for Duchenne Muscular Dystrophy: A Scoping Review.#430857NCBI PubMed Central
• Microdystrophin Gene Transfer Study in Adolescents and Children With DMD#836679ClinicalTrials.gov
• AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.#901257ClinicalTrials.gov
• Pediatric Radio Frequency Coils Generic#968723ClinicalTrials.gov
• Motor Imagery on Children With DMD on Gait and Balance Functions#994209ClinicalTrials.gov
• Integrating Genetic Modifier Genotype With Serum Proteomics in Duchenne Muscular Dystrophy Clinical Trials Links LTBP4 Genetic Modifier to IL-23/CD93 Pathways in Muscle#1001627ODU Digital Commons
• Drp1-FGF21 axis drives FAPs-mediated fibro-adipogenic degeneration in Duchenne muscular dystrophy#4935Europe PMC
• The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: Considerations for the design of clinical trials#211255IRIS
• High-throughput discovery of arginine-depleted peptides enables effective antisense delivery for Duchenne muscular dystrophy#356352bioRxiv / medRxiv
• A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)#982731ClinicalTrials.gov
• A novel approach to systemic delivery of therapies in muscular dystrophies through engineering of hematopoietic stem cells#330012HAL (France)
• Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy#159217HAL (France)
• Supplementary Material for: Outcomes of phentermine-topiramate in children living with Duchenne muscular dystrophy#192991Figshare
• Home-based exercise training in boys with Duchenne muscular dystrophy: A 12-month intervention study.#440910NCBI PubMed Central
• Transcriptomic profiling of skeletal muscle in the DMDmdx rat model of Duchenne muscular dystrophy#157654HAL (France)
• A Validated Prognostic Score for Time to Loss of Ambulation in Patients With Duchenne Muscular Dystrophy.#322980NCBI PubMed Central
• AI-powered therapeutic aptamer drug discovery: Targeting the CT-domain of CTGF for duchenne muscular dystrophy.#371674NCBI PubMed Central
• NuMA1 controls myonuclear motility in striated skeletal muscle through AMPK activity and is impaired in Duchenne muscular dystrophy#781259HAL (France)
• The U.K. NorthStar Clinical Network#967364ClinicalTrials.gov
• Residual Dp71 Expression Is Sufficient to Preserve Retinal Vascular Homeostasis in a Mouse Model of Duchenne Muscular Dystrophy#159297HAL (France)
• Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy#176853HAL (France)
• Improving Nocturnal Non-Invasive Positive Pressure Support Adherence in Children and Young Adults With Duchenne Muscular Dystrophy.#420988NCBI PubMed Central
• Disease progression and economic burden of duchenne muscular dystrophy: A retrospective study using Swedish register data.#433903NCBI PubMed Central
• MicroRNA expression profile of fibro-adipogenic progenitors in Duchenne muscular dystrophy reveals dysregulation of adipogenesis-related microRNAs#478534Europe PMC
• Screening for brain-related comorbidities in Duchenne muscular dystrophy: Construction, reliability, and validity of the BIND screener#642417Europe PMC
• Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders#804447ClinicalTrials.gov
• Prevalence and Characteristics of Cardiac Arrhythmia in Duchenne Muscular Dystrophy: A Retrospective, Single-Center Holter Electrocardiographic Monitoring Study.#265571NCBI PubMed Central
• Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy#300698Europe PMC
• Extension Study of Drisapersen in DMD Subjects#850751ClinicalTrials.gov
• Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim#894067ClinicalTrials.gov
• The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Neuromuscular Disorder#812631ClinicalTrials.gov
• A Real-World Target Trial Emulation of Eteplirsen, Casimersen, and Golodirsen to Evaluate Survival Among Patients with Duchenne Muscular Dystrophy.#483468NCBI PubMed Central
• Duchenne muscular dystrophy disease severity impacts skeletal muscle progenitor cells systemic delivery#492037eScholarship
• Transition to adulthood in Duchenne Muscular Dystrophy: a systematic review with narrative synthesis on health systems, policies, and the role of health care providers.#275757NCBI PubMed Central
• Twice Weekly Steroids and Exercise as Therapy for DMD#806220ClinicalTrials.gov
• The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD#845543ClinicalTrials.gov
• Examining the emotional impact and financial strain on caregivers of those with Duchenne muscular dystrophy in Saudi Arabia: findings from a questionnaire-based cross-sectional study.#338746NCBI PubMed Central
• Development of respiratory care guidelines for Duchenne muscular dystrophy in the UK: key recommendations for clinical practice#347986IRIS
• Dose- and genotype-dependent cardiac arrhythmia and sudden death in rats following microdystrophin gene therapy#308281HAL (France)
• The Dystrophin Glycoprotein Complex Regulates the Epigenetic Activation of Muscle Stem Cell Commitment#781257HAL (France)
• The Paradox of Life Extension with Traditional Cost-Effectiveness Analysis: A Case Study with Duchenne Muscular Dystrophy#1053060Europe PMC
• Asynchrony Related to Mechanical Ventilation Exacerbates Diaphragm Dysfunction in a Mouse Model of Duchenne Muscular Dystrophy#137618HAL (France)