Results 81 to 90 of about 21,009 (196)

Five‐Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1/2a Study

Muscle &Nerve, Volume 74, Issue 2, Page 397-403, August 2026.
ABSTRACT Aims We report 5‐year results from a phase 1/2a study of delandistrogene moxeparvovec, a recombinant adeno‐associated virus serotype rh74 vector‐based gene therapy for Duchenne muscular dystrophy (DMD), with post hoc analyses contextualizing functional outcomes. Methods Four ambulatory patients with DMD (≥ 4–< 8 years at enrollment) entered an
Jerry R. Mendell, Zarife Sahenk, Linda P. Lowes, Megan A. Iammarino, Lindsay N. Alfano, Craig M. McDonald, James E. Signorovitch, Jim Jin, Jing Li, Stefanie Mason, Louise R. Rodino‐Klapac   +10 more
wiley   +1 more source

Quantitative MRI Assessment of Myotoxin‐Induced Skeletal Muscle Damage of mdx Mice

Muscle &Nerve, Volume 74, Issue 2, Page 485-491, August 2026.
ABSTRACT Introduction/Aims Although magnetic resonance imaging (MRI) has been extensively applied in dystrophic muscle, longitudinal characterization of multiple quantitative MRI parameters during degeneration and regeneration remains limited. This study aimed to longitudinally characterize changes in quantitative MRI parameters in control and ...
Ravneet Vohra, Joshua Park, Feng Zhang, Jeffrey S. Chamberlain, Donghoon Lee   +4 more
wiley   +1 more source

Basal Energetics and Phosphocreatine Recovery Kinetics in Ambulatory Boys With Duchenne Muscular Dystrophy

NMR in Biomedicine, Volume 39, Issue 8, August 2026.
Basal energetics and phosphocreatine (PCr) recovery kinetics of the lower leg anterior compartment (primarily tibialis anterior; TA) were evaluated at rest and after dorsiflexion muscle contractions in ambulatory boys with Duchenne muscular dystrophy (DMD) and unaffected controls using 31P‐MRS.
Pratiksha P. Awale, Christophor Lopez, Tanja Taivassalo, Krista Vandenborne, Glenn A. Walter, Sean C. Forbes   +5 more
wiley   +1 more source

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

Developmental Medicine &Child Neurology, Volume 68, Issue 8, Page 1080-1096, August 2026.
The Brain Involvement iN Dystrophinopathies (BIND) screener is an 18‐item questionnaire with strong reliability and validity for identifying potential brain‐related comorbidities in Duchenne muscular dystrophy. It allows rapid, cross‐age and cross‐country screening for both clinical and research purposes, demonstrating good sensitivity and specificity.
Ruben Miranda, Pien M. M. Weerkamp, Anna Kolesnik, Chloe Geagan, Daniela Chieffo, Mariana Suárez‐Bagnasco, David Skuse, Elizabeth Vroom, Erik H. Niks, John Vissing, Isabelle Desguerre, Volker Straub, Francesco Muntoni, Eugenio Mercuri, Jos G. M. Hendriksen, on behalf of BIND Consortium, Nathalie Angeard, Dimitrios Athanasiou, Suzie‐Ann Bakker, Nathalie Boddaert, Ellie Drummond, Chloe Durrlemann, Fernanda Fortunato, Luis Miguel García‐Moreno, Mattia Gervasi, Rosanne Govaart, Irina Guliaeva, Catherine Hankinson, Leslie Hemar, Rebecca Hendel, Hermien Kan, Leevi Kerkela, Gregory Landon, Monika Malinova, William Mandy, Federica Moriconi, Monia Pellizzari, Sarah Poncet, Elvina Sakellariou, Sergiu Siminiuc, Anna Würgler Slipsager, Lily Smythe, Pietro Spitali, Mads Peter Godtfeldt Stemmerik, Asmus Vogel, Jeanne Wolstencroft, Camilla Zanetti   +46 more
wiley   +1 more source

Resistance‐exercise‐induced stress intervenes in TGF‐β signaling by cooperatively downregulating nuclear αB‐crystallin and SMAD4 in human skeletal muscle fibers

The FEBS Journal, Volume 293, Issue 15, Page 4624-4644, August 2026.
Alpha‐crystallin B chain (CRYAB) has been reported to stabilize mothers against decapentaplegic homolog 4 (SMAD4) in transforming growth factor‐β (TGF‐β) signaling, enabling target gene transcription. We show nuclear CRYAB–SMAD4 interaction for the first time in human skeletal muscle fibers and its regulation by exercise.
Kirill Schaaf, Cassandra Duda, Tihomir Kostov, Patrick Diel, Käthe Bersiner, Wilhelm Bloch, Sebastian Gehlert, Daniel Jacko   +7 more
wiley   +1 more source

A high‐fat, high‐sucrose diet exacerbates muscle and metabolic pathology and undermines glucocorticoid efficacy in dystrophin‐deficient mice

The Journal of Physiology, Volume 604, Issue 15, Page 6440-6464, 1 August 2026.
Abstract figure legend A high‐fat, high‐sucrose diet (HFHSD) exacerbated muscle and metabolic dysregulation in mdx mice while also altering the physiological response to prednisolone (Pred) In diaphragms of control diet (CD)‐fed mdx mice, prednisolone treatment improved muscle function and reduced fibrosis, inflammatory signalling, and mitochondrial ...
Morgan E. Vorwald, Swathy Krishna, Ji Heun Lee, Melissa Roths, Rudy J. Valentine, Joshua T. Selsby   +5 more
wiley   +1 more source

Myonuclear Dynamics After Skeletal Muscle Surgical Injury

The FASEB Journal, Volume 40, Issue 14, 31 July 2026.
Using a recombination‐independent doxycycline inducible model of timed nonsatellite cell‐derived myonuclear labeling in adult skeletal muscle, we show that resident myonuclei move and become displaced within intact muscle fibers after muscle surgical injury.
Micah Goeke, Nathan Serrano, Pieter Jan Koopmans, Kevin A. Murach   +3 more
wiley   +1 more source

Utrophin Compensates dystrophin Loss during Mouse Spermatogenesis

Scientific Reports, 2017
Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder resulting from mutations in the dystrophin gene. The mdx/utrn −/− mouse, lacking in both dystrophin and its autosomal homologue utrophin, is commonly used to model the clinical symptoms of
Hung-Chih Chen, Yu-Feng Chin, David J. Lundy, Chung-Tiang Liang, Ya-Hui Chi, Paolin Kuo, Patrick C. H. Hsieh   +6 more
doaj   +1 more source

Immunologic investigations into transgene directed immune-mediated myositis following delandistrogene moxeparvovec gene therapy

Scientific Reports
Delandistrogene moxeparvovec is an rAAVrh74 vector-based gene transfer therapy that delivers a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered, functional form of dystrophin shown to stabilize or slow disease progression ...
Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac   +14 more
doaj   +1 more source

A combinatorial oligonucleotide therapy to improve dystrophin restoration and dystrophin-deficient muscle health

Molecular Therapy: Nucleic Acids
Despite the proven safety of dystrophin-targeting phosphorodiamidate morpholino oligomer (PMO) therapy, poor delivery of the PMOs limit the efficacy of this dystrophin restoring gene therapy for Duchenne muscular dystrophy (DMD).
Young Jae Moon, Ravi Hindupur, Iteoluwakishi H. Gamu, Nikki M. McCormack, Fatima Shaikh, James S. Novak, Jyoti K. Jaiswal   +6 more
doaj   +1 more source