In a study supported by the National Institute of Health (NIH), researchers at Iowa State University found that a drug called A15/283 helped to ameliorate symptoms of a mild form…
The heart-breaking story of baby Charlie Gard, who recently died of a rare genetic condition called encephalomyopathic mitochondrial DNA depletion syndrome, touched the lives of families around the world. Although…
This month, Soliris®, a treatment produced by Alexion Pharmaceuticals, Inc. was approved by the European Commission for the treatment of Refractory Generalized Myasthenia Gravis (gMG). Myasthenia gravis (MG) is a…
If you're looking for a time table for adjusting to, and accepting, the diagnosis of a rare disease, you won't find one, because there isn't one. The variety of rare…
Duchenne muscular dystrophy (DMD) is a heartbreaker for the the 250,000 families worldwide whose sons are born with this debilitating, progressive disease. It is rare, and yet the ripples of its…
Cancer. This word can inspire fear in even the most stalwart of individuals. How many other diseases are known by a phrase involving just the first letter? The Big C.…
The first Disorder: The Rare Disease Film Festival (RDFF) is coming to Boston, MA this October! Check out details below: Who: Rare Outreach Coalition, LLC. What: Disorder: The Rare Disease Film…
At 54 years of age, Kathleen was healthy and physically fit. She knew about her grandfather’s death at the age of 30 to a heart attack. She also knew about…
Diamond Blackfan anemia (DBA) occurs when the bone marrow in one’s body either does not produce red blood cells or doesn't create enough of them. Sometimes called Blackfan Diamond anemia,…
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