Nancy A. Melville November 06, 2023 PHOENIX — Children with Duchenne muscular dystrophy (DMD) treated with the only gene therapy to date to be approved for treatment of disease in the United States show sustained maintenance of motor function after 4 years, compared with untreated patients who showed significant decline over the same time period, new research...
Tag: <span>muscular dystrophy</span>
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Researchers target protein that can slow down muscular dystrophy
by Laurie Fickman, University of Houston Graphical abstract. Credit: JCI Insight (2023). DOI: 10.1172/jci.insight.164768 A team of researchers at the University of Houston College of Pharmacy is reporting that by manipulating TAK1, a signaling protein that plays an important role in development of the immune system, they can slow down disease progression and improve muscle function in Duchenne muscular dystrophy...
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Could a novel small molecule slow or reverse the effects of Duchenne muscular dystrophy?
by Wiley Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle fibers by adipose cells. Credit: Wikimedia Commons/Public Domain In a new study published in The FASEB Journal, investigators demonstrated the potential of a molecule that may help overcome some of the...
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Toxic protein linked to muscular dystrophy and arhinia
by National Institutes of Health Credit: Pixabay/CC0 Public Domain Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For patients who have facioscapulohumeral muscular dystrophy (FSHD), or a rare facial malformation called arhinia,...
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Cancer drug shows potential as treatment for muscular dystrophy
by University of British Columbia Effects of CSF1R inhibitors – and the absence of self-renewing resident macrophages – on muscle regeneration in mice. Credit: F. Babaeijandaghi, et al., Science Translational Medicine (2022) Researchers at the University of British Columbia’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular...
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Study reveals how Duchenne muscular dystrophy causes heart rhythm problems
ELIFE IMAGE: STEM CELL-DERIVED CARDIOMYOCYTES PRODUCED TO STUDY ARRHYTHMIC MECHANISMS CREDIT: FÁTIMA LOIS The results help explain why as many as 60% of patients with DMD have potentially life-threatening heart rhythm abnormalities. They may also suggest potential treatment strategies for heart problems in people with DMD. Mutations in a gene that encodes a muscle-protecting protein...
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Muscle stem cell technology a prelude to new muscular dystrophy therapeutics
by Jim Fessenden, University of Massachusetts Medical School Isolation and characterization of iMyoblasts. (A) Schematic of a three-stage transgene-free iPSC induction, iMyoblast reserve cell isolation, and iMyotube differentiation protocol. Images of S2 cells and iMyoblasts immunostained with MYOD1 antibody, and S3 iMyocytes and iMyotubes immunostained with MF20 myosin antibody. Nuclei are stained with DAPI. Scale...
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Cellular therapy improves signs and symptoms of Duchenne muscular dystrophy
by UC Davis Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle fibers by adipose cells. Credit: Public Domain A clinical trial at UC Davis Health and six other sites showed that a cellular therapy offers promise for patients with late-stage...
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New treatment could benefit up to 45 percent of patients with Duchenne muscular dystrophy
by Gillian Rutherford, University of Alberta Toshifumi Yokota led research to develop and test a new “cocktail” drug that may be beneficial for up to 45 per cent of patients with a common form of muscular dystrophy — a significantly higher proportion than currently available treatments. Credit: University of Alberta Up to 45% of patients...
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Steroid treatments for Duchenne muscular dystrophy may depend on the clock
by Cincinnati Children’s Hospital Medical Center In mice, prednisone enhances muscle activities when once-weekly doses are given at the beginning of the day but not at the beginning of the night cycle. Credit: Cincinnati Children’s Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness...