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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(HealthDay)—Its extremely high price tag means that a lifesaving medication to treat young children with spinal muscular atrophy is simply too expensive for most families.
Jul 30, 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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Gene therapy—for so long something that belonged to the future—has just hit the streets.
Jun 5, 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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(HealthDay)—The first gene therapy has been approved to treat children younger than 2 years with spinal muscular atrophy (SMA), the U.S. Food and Drug Administration announced Friday.
May 28, 2019
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U.S. regulators have approved the most expensive medicine ever, for a rare disorder that destroys a baby's muscle control and kills nearly all of those with the most common type of the disease within a couple of years.
May 24, 2019
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There is now further evidence that a drug that is effective in treating the rare muscle-wasting disease spinal muscular atrophy (SMA) early in life may be associated with improvement in older children, according to a study ...
Apr 24, 2019
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