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Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Treatment with Soliris (eculizumab) provided adults with generalized myasthenia gravis (gMG) clinically meaningful improvements through three years in daily living, muscle strength, functional ability and quality of life, according to early data from an extension study. Lower rates of MG exacerbations and hospitalizations were also observed. The…

The inner ear function in myasthenia gravis (MG) patients is significantly impaired, and hearing tests can serve as a diagnostic and monitoring tool, scientists from South Korea say. Their study, “Abnormalities of Otoacoustic Emissions in Myasthenia Gravis: Association With Serological and Electrophysiological Features” was published in the…