Vamorolone offers new treatment option with fewer side effects Business Announcement CHILDREN’S NATIONAL HOSPITAL WASHINGTON (October 27, 2023) – Boys with Duchenne muscular dystrophy (DMD) have a clinically proven, new treatment option with the Food and Drug Administration’s approval Thursday of vamorolone, a steroidal-type, anti-inflammatory drug developed based on research performed at Children’s National Hospital. Created...
Tag: <span>dystrophy</span>
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New Duchenne muscular dystrophy drug shows benefit in clinical trial
by Duke University Medical Center A new drug offers hope for young boys with the progressive neuromuscular disease Duchenne muscular dystrophy (DMD) by potentially offering an alternative to high-dose glucocorticoids that have significant side effects. Interim results from a 24-month clinical trial at Duke Health and other institutions suggest that the drug, vamorolone, may retain or...
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Scientists uncover a novel approach to treating Duchenne muscular dystrophy
SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE Scientists at Sanford Burnham Prebys Medical Discovery Institute, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring (fibrosis). The study,...
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Drugging the undruggable: A treatment path for muscular dystrophy
by Yale University Credit: CC0 Public Domain Researchers at Yale have identified a possible treatment for Duchenne muscular dystrophy (DMD), a rare genetic disease for which there is currently no cure or treatment, by targeting an enzyme that had been considered ‘undruggable.’ The finding appears in the Aug. 25 edition of Science Signaling. DMD is the most...
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RNA scientists advance early detection of Duchene muscular dystrophy
by University at Albany The devastating effects of Duchene muscular dystrophy (DMD) can be mitigated if detection takes place in early childhood. Sadly, early diagnosis is rare. Now, University RNA researchers have discovered a novel method that could solve this long-standing problem. The research team, from the UAlbany laboratories Professor Igor Lednev of Chemistry and...
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Scientists edge closer to treatment for myotonic dystrophy
by University of Nottingham untreated and treated with kinase inhibitors. Following treatment with inhibitors targeting CDK12 nuclear foci in DM1 cells are reduced. Credit: Dr. Ami Ketley , the University of Nottingham Scientists at the University of Nottingham have taken a step closer towards developing a treatment for the long-term genetic disorder, myotonic dystrophy. In...
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Gene therapy for Duchenne muscular dystrophy safely preserves muscle function
by Perelman School of Medicine at the University of Pennsylvania A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large animal models, according to a first-of-its-kind study from Penn Medicine researchers. The...
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Q&A: Understanding Duchenne muscular dystrophy
by From Mayo Clinic News Network, Mayo Clinic News Network Dear Mayo Clinic: What is Duchenne muscular dystrophy, and what causes it? Is treatment available? Can Duchennemuscular dystrophy be cured? A: As with all forms of muscular dystrophy, Duchenne muscular dystrophy is caused by a genetic defect. The defect leads to muscle weakness and loss of muscle mass that worsens over time. Medication and physical therapy are used to...