Medical research
Death in US gene therapy study sparks search for answers
The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him.
Nov 4, 2022
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Muscular Dystrophy
Medical research
Scientists studying fatal muscle wasting disease make significant discovery
Duchenne muscular dystrophy (DMD) is caused by a genetic mutation and affects one in every 5,000 boys born. Because the affected gene is on the X chromosome, girls are carriers of the mutant gene but develop the disease only ...
Sep 27, 2022
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Medications
Blocking an ion channel improves muscle function and survival in mice with severe Duchenne muscular dystrophy
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne ...
Sep 13, 2022
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14
Medications
Vamorolone effective, safe for boys with Duchenne muscular dystrophy
For boys with Duchenne muscular dystrophy (DMD), vamorolone, a structurally unique dissociative steroidal anti-inflammatory drug, is effective and safe over a 24-week period, according to a study published online Aug. 29 ...
Sep 1, 2022
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Health
Expert explains when swallowing issues are more than an accident
Many people have experienced the discomfort of food or a beverage accidentally going to the wrong place when swallowing. But swallowing issues sometimes become chronic and may be a sign of a health condition that should be ...
Aug 24, 2022
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36
Medical research
Cancer drug shows potential as treatment for muscular dystrophy
Researchers at the University of British Columbia's School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.
Jun 29, 2022
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80
Diseases, Conditions, Syndromes
Study reveals how Duchenne muscular dystrophy causes heart rhythm problems
Abnormalities in the proteins responsible for transmitting electrical signals in the heart likely cause abnormal heart rhythms in patients with Duchenne muscular dystrophy (DMD), shows a study published in eLife.
Jun 28, 2022
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17
Medications
Study finds daily steroids safe and slow the progression of Duchenne muscular dystrophy
New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated. University of Rochester Medical Center (URMC) ...
May 4, 2022
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9
Diseases, Conditions, Syndromes
Cell treatment slows disease in Duchenne muscular dystrophy patients
A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles—including the heart muscle—in Duchenne muscular dystrophy patients, a new study published in the international ...
Apr 13, 2022
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Overweight & Obesity
Can weekly prednisone treat obesity?
Obese mice that were fed a high-fat diet and that received prednisone one time per week had improved exercise endurance, got stronger, increased their lean body mass and lost weight, reports a new Northwestern Medicine study. ...
Apr 1, 2022
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