New drug shows potential in treating Duchenne muscular dystrophy
A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.
Dec 16, 2024
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Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Muscular Dystrophy, Duchenne
MeSH tree: C16.320.577.300
Researchers discover zip code that allows proteins to hitch a ride around the body
Researchers at The Ottawa Hospital and the University of Ottawa have discovered an 18-digit code that allows proteins to attach themselves to exosomes—tiny pinched-off pieces of cells that travel around the body and deliver ...
Dec 11, 2024
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Evaluating long-term survival and cardiac efficacy of a gene therapy for Duchenne muscular dystrophy
A recent study published in the journal Human Gene Therapy evaluated the long-term survival and cardiac efficacy of the gene therapy delandistrogene moxeparvovec in a rat model of Duchenne muscular dystrophy (DMD).
Dec 4, 2024
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$3.2 million per dose Elevidys fails to meet primary endpoint in phase 3 trial
A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings published in Nature Medicine. A News ...
Preventing skeletal muscle fatigue in Duchenne muscular dystrophy through cell therapy
Duchenne muscular dystrophy (DMD) is a neuromuscular disease causing progressive skeletal muscle weakness and fatigue. The research team led by Associate Professor Hidetoshi Sakurai (Department of Clinical Application), Researcher ...
Oct 7, 2024
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Next-gen gene therapy vector for muscle diseases uses AI predictive methodology to improve efficacy and safety
A new study describes the use of artificial intelligence (AI) in designing a new generation of capsids, the structures that envelop genetic material of adeno-associated viruses (AAV), to improve gene therapies for muscle ...
Sep 12, 2024
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Detective algorithm predicts best drugs for genetic disorders and cancer
A computational model built by researchers at the Institute of Research in Biomedicine (IRB Barcelona) and the Centre for Genomic Regulation (CRG) can predict which drugs will be most effective in treating diseases caused ...
Aug 22, 2024
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Promising new screening tool may help manage neurobehavioral difficulties in children with Duchenne muscular dystrophy
A pilot study using a new screening tool shows promise for assessing neurobehavioral difficulties in children with Duchenne muscular dystrophy (DMD) and improving quality of life for patients and their families. Results appear ...
Aug 14, 2024
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New gene therapy approach shows promise for Duchenne muscular dystrophy
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people ...
Jul 25, 2024
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Skeletal muscle regeneration discovery paves the way for targeted therapies for muscle disorders
Recent research from the University of Houston College of Pharmacy identifies key mechanisms of skeletal muscle regeneration and growth of muscles following resistance exercise. It's a finding that opens the door to the development ...
Jul 22, 2024
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