The causes of rare diseases could be uncovered using an approach created to identify genetic mutations that trigger a muscle-wasting condition, a study suggests.
Dec 18, 2019
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(HealthDay)—In a head-to-head study of mineralocorticoid receptor antagonists, spironolactone was found to be noninferior to eplerenone for slowing the progression of heart damage in boys with Duchenne muscular dystrophy ...
Oct 28, 2019
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A gene therapy being developed at Penn Medicine to treat Duchenne muscular dystrophy (DMD) successfully and safely stopped the severe muscle deterioration associated with the rare, genetic disease in both small and large ...
Oct 7, 2019
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A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy (DMD).
Sep 25, 2019
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About one in 8,000 people have facioscapulohumeral muscular dystrophy, according to a 2014 study, which is relatively common in the world of genetic diseases. New University of Minnesota Medical School research identifies ...
Sep 11, 2019
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A team at Fred Hutchinson Cancer Research Center has identified a gene that could make immunotherapy treatments, specifically checkpoint inhibitors, work for a wider variety of cancer patients. The study, published today ...
Jul 18, 2019
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Duchenne muscular dystrophy occurs in boys and is characterized by progressive muscle degeneration and weakness leading to a decline in respiratory function. Strategies to arrest this severe progressive deterioration are ...
Jul 9, 2019
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Standing up from a chair, climbing stairs, brushing one's hair—all can be a struggle for people with a rare form of muscular dystrophy that causes progressive weakness in the shoulders and hips. Over time, many such people ...
Jun 3, 2019
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Dear Mayo Clinic: What is Duchenne muscular dystrophy, and what causes it? Is treatment available? Can Duchenne muscular dystrophy be cured?
May 30, 2019
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Isabelle Richard's team, a CNRS researcher in an Inserm unit at Genethon, the AFM-Telethon laboratory, has demonstrated the efficacy of gene therapy and determined the effective dose for treating a rare muscle disease, gamma-sarcoglycanopathy, ...
May 27, 2019
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