MeSH tree: C16.320.577.300
In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid screening tool to help identify individuals with Duchenne muscular dystrophy (a neuromuscular disorder) ...
Jan 14, 2026
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Until relatively recently, children and young people with life-shortening conditions were not expected to survive into adulthood.
Jan 7, 2026
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The United States on Friday said it would add its most serious warning label to a gene therapy treatment for a debilitating muscle-wasting disease, and also limit its approved use.
Nov 15, 2025
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If cancer is a disease of overabundance, where cells divide without restraint and tumors grow despite the body's best interests, then degenerative diseases are disorders of deprivation. When malfunctions occur in the biological ...
Oct 30, 2025
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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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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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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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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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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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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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