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…
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,…
Happy Back-to-School Week Patient Worthians! If you're going back to school or your children are going back to school, we know it's a hectic and fun time of year. So…
When I was a kid, pedal power was the way to go. In fact, it was the only way to go—anywhere. It was a mode of transportation for us. We…
In recognition of Spinal Muscular Atrophy Awareness Month, Joe Akmakjian, an SMA warrior/blogger, is promoting a unique understanding of his rare disedsase. Originally published on the Muscular Dystrophy Association's Strongly…
Mended Hearts is a nonprofit group that helps bring hope to heart disease patients, caregivers and families. In Alabama specifically, the local Mended Hearts group had not been active for…
One night in March 2016,13-year-old Treyvon Summers experienced extreme pain that woke him up out his sleep. This was the start of him losing his mobility in both his legs.…
Whenever I feel the stress of life getting to me, I hop over to YouTube and search “random acts of kindness” or “heartwarming acts.” Some of the results? The police…
Sickle cell disease, hemophilia, and thalassemia are a few of the inherited blood disorders. Dr. Waveney Charles is a hematologist, or specialist in blood-related conditions. She believes that the number…
According the Cystic Fibrosis News Today, when Claire was first born, she was only expected to live to the age of 10. Then Claire’s life expectancy lengthened to 18, and…
Among the many uses of social media, medical diagnosis for rare diseases, for everything from acromegaly to Zellweger spectrum syndromes, may not be the first that comes to mind. But…
A recent survey of more than 150 idiopathic pulmonary fibrosis (IPF) patients demonstrates the invisible burden the rare disease causes. It is no secret that any rare disease has the…
While there is good scientific evidence backing up the fact that omega-3 fatty acids found in fish oil can help slow or even prevent heart disease, but does fish oil…
Learn more about acromegaly at the most low maintenance seminar of all time—on your own computer! From 1:00PM-2:00PM EST on Wednesday, August 23, 2017, sign up to hear Dr. Adriana G.…
October is a time for celebrating autumn and Halloween in the US. But it's also Gaucher Disease Awareness Month! Mark your calendars for this Gaucher Disease event: Who: The National…
I am a firm believer that amidst all the fads that come and go, there is one trend that will always be popular. This, of course, is the do-it-yourself craze…
Damien took a lot of naps. I mean, a lot! It’s true; he was a very active Doberman Pinscher. He fetched everything—tennis balls, Frisbees, shoes that hadn't been put away—you…
As autoimmune disorders become an increasing problem throughout the world, particularly in developed countries, there has been an increasing amount of research dedicated to finding a cure, and the role…
The Familial Hypercholesterolemia Foundation's main mission is to raise awareness of familial hypercholesterolemia (FH) to the public. As part of this goal, the FH Foundation annually hosts Familial Hypercholesterolemia Awareness…
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