Neuromuscular disease care is entering a new era of hope and innovation
Every movement we make, whether walking, speaking or even breathing, depends on the health of our muscles and nerves.
Sep 19, 2025
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Musculoskeletal Diseases: News and Research on Muscular Dystrophy, Duchenne
MeSH tree: C05.651.534.500.300
Under Trump, FDA seeks to abandon expert reviews of new drugs
FDA leaders under President Donald Trump are moving to abandon a decades-old policy of asking outside experts to review drug applications, a move critics say would shield the agency's decisions from public scrutiny.
Sep 15, 2025
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Media visibility and costs of new medicines under examination—what does the media highlight?
A study conducted by the University of Eastern Finland and Fimea found that the Finnish news media has given GLP-1 agonists used to treat obesity, such as semaglutide, a great deal of attention while many other new medicines ...
Sep 10, 2025
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Cell therapy prevents damage from Duchenne muscular dystrophy in mouse model
A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular dystrophy, according to new research from the Smidt Heart Institute at Cedars-Sinai.
Aug 4, 2025
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FDA vaccine chief steps down after controversial tenure
The U.S. Food and Drug Administration's (FDA) top vaccine official is stepping down after just three months in a role that upset drug companies, patient groups and some political leaders.
Jul 31, 2025
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Novel approach enhances neuromuscular function in patients with Duchenne muscular dystrophy
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated that inhibiting GLUD1 ...
Jul 16, 2025
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New route into cells could make gene therapies safer
Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular ...
Jul 15, 2025
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High hopes and high costs: Exploring advances in Duchenne muscular dystrophy gene therapy
To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec, the American Academy of Neurology (AAN) has issued ...
May 14, 2025
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Dr. Vinay Prasad to head FDA vaccine, biologics division
The U.S. Food and Drug Administration (FDA) has chosen Dr. Vinay Prasad, a professor at the University of California-San Francisco, to lead its Center for Biologics Evaluation and Research.
May 7, 2025
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Existing treatments may help fight symptoms of severe form of muscular dystrophy
A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help control chronic muscle inflammation in Duchenne muscular dystrophy (DMD), a severe condition that ...
Apr 30, 2025
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