It takes an average of seven years for a patient with a rare disease like severe combined immunodeficiency (SCID) to receive a correct diagnosis; and that comes after the patient…
If you have acromegaly, you know that available treatment options are very limited, so when Chiasma, a pharmaceutical company based in Waltham, Massachusetts, received notification that the FDA was not…
If you are trying to dive into aplastic anemia, here are some interesting facts from the Seattle Children’s Hospital that you should definitely check out. First, for anyone who’s been…
Those of us with Gaucher disease who know how much uncertainty, fear, and anger enzyme replacement treatment (ERT) shortages cause, REJOICE! Researchers have found that the oral substrate reduction therapy…
Usted no va a venir a través de demasiadas personas en sus vidas como Elaine Gómez y su hija, Michelle DeMont. Y es probable que no se encontrará con el…
Could the study of Alzheimer's disease lead to treatments for GLUT1 deficiency syndrome? It's been known for a while that there's a link between Alzheimer's and how the brain metabolizes…
If you're living with thrombocytopenia (that is, a severely low level of platelets in your blood), then Dr. Mei X. Wu may be your new best friend. The associate professor…
Amidst the hype leading up to the first head-to-head American Presidential debate with Trump vs. Clinton, the topic of Venezuela’s drug shortage of aplastic anemia drugs wasn't mentioned. But having…
Are you a guy who'd rather suck lemons than talk about your Sjogren's syndrome? Men who are living with Sjogren’s syndrome are sometimes forgotten because the disease mostly affects women…
Describing your acromegaly isn’t easy. I’m sure you’ve been met with enough blank stares or uncomfortable jokes about “giants” to last a lifetime. Fact is, sometimes people will not get…
De acuerdo, Dr.House: Tenemos padres por primera vez que afirman su recién nacido es "irritable, inquieto, vómitos explosivos, y se niega a comer." Aunque también decir que el niño tiene…
Uno pensaría que en el mundo actual de la tecnología, estaríamos delante de la curva, especialmente cuando se refiere a las pruebas de diagnóstico / pronóstico. Lo que pasa descubrimiento…
En este momento, usted y yo estamos justo en el medio de uno de los más grandes de la historia las edades de información. Tenemos más formas de aprender sobre…
Happy Weekend Before the Election Patient Worthians! Get some rest, Tuesday is going to be a big one in the United States. But while you're hanging out, check out these…
Cystinosis is a metabolic disease in which the amino acid cystine crystallizes and then begins to accumulate in the kidneys, eyes, liver, and white blood cells. Children are typically diagnosed…
First of all, many people have experienced a nose bleed at some point. A nose bleed is a very common annoyance for many. These people typically just brush it off as something…
On the surface, it seems self-evident that social collaboration can have a positive impact--especially when it comes to Duchenne muscular dystrophy (DMD). Working together with all invested participants to influence…
I am completely BAFFLED About Addison’s disease! Are you?! You Should be! It’s Monday night, and I’m sitting—rather, lounging on the sofa, feeling baffled after reading a post from a…
Para los padres de niños con distonía, que la frustración es una realidad horrible, en curso. Los padres como Melissa Phelps de Gallaten, Kentucky lo sabe muy bien hijas-Madison y…
In my humble opinion, the Urology Care Foundation has set the standard when it comes to providing factual information about Cushing’s syndrome (CS). And if you or your loved one…
2016 has been a very bizarre, upsetting year. As an American, I can’t wait until I can bury most everything about 2016 in the back corner of the history books.…
I always have a knee-jerk reaction that borders on anger when I read about babies who are dealt an unfair hand in the rare disease world. It seems incredibly unfair…
Have you ever fainted? I have. Three times and all were related to receiving shocking news. The first time was when my grandmother unexpectedly passed away. The second time was…
Legend has it that Archimedes once ran through the streets of Syracuse naked yelling, “Eureka!” because he had discovered an amazing scientific principle. (BTW, if you're thinking, "What on earth…
The days of summer are at a close. The temperature has finally dropped, and there’s a chill in the air. A new season is upon us. Finally! School is back…
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