The only time we actually like the FDA is when they finally approve drugs—especially when they're for rare diseases. Well, in April of 2016, they made a rare group of…
Welcome to this week's Editor's Choice! When you have a rare disease like dystonia, you come across a ton of different reactions from people in regards to your illness. One…
Featured Image is by Gavin Whitner at musicoomph.com. Dude, I’m sorry but this narcolepsy video needs way more than 90-some views. Posted on Grey Alquist’s YouTube channel, two young man made…
When it comes to science and medicine, big things seldom come in big packages (get your minds out of the gutter). Groundbreaking discoveries that change our understanding of a disease…
dedo mutilado? contusión ósea severa? Fractura en la espalda? No, esto no es un catálogo de las lesiones del club de la lucha, éstas son algunas lesiones reales el Miami…
Most people know that high cholesterol is a ticking time bomb, and that it’s important to eat heart healthy foods low in saturated fat and cholesterol. Public health awareness—and the…
Kim Pimley is a mover and shaker, a businesswoman on the go. She’s one of those energetic people you see running up an escalator, to save time, and also, just…
With rare disease, everyone is familiar with the concept that early diagnosis typically means better patient outcomes. Such is the case with acromegaly. According to a recent article in healio,…
Kallman syndrome is a condition in which puberty is delayed or completely absent from the individual. According to the NIH, it effects just one out of every 10,000 to 86,000…
Hipercolesterolemia familiar heterocigótica (o HeFH), nunca has oído hablar de él? Es un bocado. Es también una condición que hace que los niveles de colesterol de alta peligrosidad y, si…
Carcinoid tumors are rare and most often occur in the lungs and intestinal tract. When they secrete chemicals into the blood stream, the follow symptoms may occur: skin flushing, facial…
According to a recent study, patients who were previously treated for acromegaly were at an increased risk for bone fractures after being treated with growth hormones for six years. Scientists at VU…
The Dystrophic Epidermolysis Bullosa Research Association of America (Debra) describes EB as "The Worst Disease You've Never Heard Of". It's a painful disease without a cure and it's rarity makes…
Emma Gaul’s story is a good reminder of how quickly life’s plans can change when your body turns against itself. Less than two years ago she was a vibrant and…
As someone who went through long-term antibiotic treatment, I can speak to the tremendous financial strain it had on my family. Well, if you live in Massachusetts, new legislative action…
Glucosa. Esto significa que el azúcar, ¿verdad? Está en su cuerpo, ¿verdad? Pero, ¿cómo se transportan y cómo sabe que su cuerpo la cantidad que necesita para el transporte? Lo…
And now for something completely different… It gives me great pleasure to present my list of VIII (a.k.a. eight) things you have to wait for…and ONE very big one you…
Si usted es una de las pocas personas en EE.UU. que viven con el síndrome de quilomicronemia familiar (FCS por sus siglas en ingles), lo más probable es que usted…
Welcome to this week's Editor's Choice! Those with acromegaly have a few options for treatment. Pituitary surgery and clinical trials for new drugs are two of these acromegaly treatment options,…
Hunter Syndrome, is a heartbreaking twist of genetic fate, and we celebrate that the FDA has granted Rare Pediatric Disease Designation to REGENXBIO's RGX-121 Gene Therapy. Hunter Syndrome causes seemingly…
Is it just me, or is EVERYBODY talking about vitamin D deficiencies these days? Suddenly, everyone I know is coming back from their health check-ups with prescriptions to combat their…
Q: What does an autoimmune disease that primarily affects tear glands and salivary glands have in common with a James Clavell novel? A: Nothing, really—other than the fact that the…
Jake Krahe, un joven de Montville Township, Ohio, ha estado viviendo con una enfermedad rara desde que tenía 19 meses de edad informa 19 action news. El diagnóstico específico es la…
If you are a rare disease sufferer in the midwest, then you should know about the US Pain Foundation's Pain Treatment Expo in August. So why go? You may find…
When people hear the word “Parkinson’s disease” (PD), they are likely to conjure an image of trembling and unsteady gait. But Parkinson’s disease has many manifestations that strike individuals unequally…
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