News tagged with rare diseases

Should we limit spending on lifesaving drugs?

"It was the best of times, it was the worst of times" is a familiar quote from the opening of Charles Dickens' "A Tale of Two Cities," but the phrase is also applicable to the specialty drug market in the United States today.

Jul 06, 2017
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What percentage of ALS is genetic?

Up to 90 percent of people with amyotrophic lateral sclerosis (ALS) report that they have no family history of the disease. Now, new research has found approximately 17 percent of such ALS cases may be caused by a gene mutation, ...

Jun 21, 2017
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Diagnosing GAN one strand at a time

Giant axonal neuropathy (GAN) is an extremely rare genetic disease with only 73 known patients worldwide. GAN causes a gradual failure of motor neurons that stimulate muscles and eventually failure of sensory neurons. New ...

May 22, 2017
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