Why disabled young people with life-shortening conditions need better support for intimacy
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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Disease or Syndrome: Muscular Dystrophy, Duchenne
Medical news and research articles
US narrows use of muscle-wasting therapy after teen deaths
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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Blocking a key protein can double yield and increase potency of muscle-regenerating cells
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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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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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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