Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing approach intended to ...
Jan 7, 2025
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A new study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its partner protein, dystrobrevin, offering new pathways for understanding and treating Duchenne Muscular ...
Jan 2, 2025
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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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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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A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1—the most prevalent form of adult-onset muscular dystrophy—provides insight into why patients develop fatty liver disease and display hypersensitivity ...
Oct 24, 2024
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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 ...
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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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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The Paris 2024 Paralympic Games has a record number of broadcasters in more than 160 countries and territories. Viewing figures are expected to top the Tokyo 2020 Games, which was shown in 154 countries and attracted 4.1 ...
Sep 3, 2024
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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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