This week, WorldSymposia™ is having their 13th Annual WORLDSymposium in San Diego! The goal of the symposium is to help researchers build a better understanding around lysosomal disease diagnostics, areas that…
On January 8, 2017, a man with one of the most brilliant minds of our age celebrated his 75th birthday. This, by itself, wouldn't be particularly remarkable, however the celebrant…
One of the most frustrating aspects of living with a chronic or rare condition is the financial stress it places on patients and their families. Doctor’s visits and prescription drugs…
Después de ser diagnosticado con una enfermedad rara, a menudo hay un sinfín de preguntas que siguen: ¿Qué voy a hacer? ¿Qué es esta enfermedad? ¿Es curable? Y la pregunta…
Hereditary angioedema (HAE) causes unexpected swelling in the hands, feet, abdomen, and face. HAE can be fatal when the patient has a laryngeal attack, where swelling in the throat makes it impossible…
You can give without loving, but you cannot love without giving. -Amy Carmichael Don't Forget-- It's also National Donor Day. Give one of the ultimate gifts of love this Valentine's…
I always love to read a heart-warming story. To me there is nothing greater than when family comes together during a hard time. That is exactly what two brothers did.…
The first medication to combat spinal muscular atrophy (SMA) has recently been approved by the United States Food and Drug Administration. It is approved for use by children as well…
12-year-old Sarah Stone has had sickle cell anemia for as long as she can remember. She experiences a lot of pain, takes lots of medicines and has had many surgeries.…
Carcinoid tumors usually develop in the gastrointestinal tract or the lungs. Because they are very slow-growing, patients may remain symptom-free for a long period of time. While this is good,…
In the rare disease world, getting a correct diagnosis can be akin to playing the lottery. Symptoms often mimic other conditions, and many healthcare professionals have never seen a patient…
New therapies have increased the likelihood of a longer life with cystic fibrosis (CF). A few decades ago, it was not common for children diagnosed with CF to live past…
Does the name Ludwig van Beethoven ring a bell? It should. He was one of the greatest composers the world has ever seen. He also suffered from cirrhosis of the liver, infectious…
We want to celebrate our patient contributors and writers for their efforts in helping the rare community, bringing awareness, understanding and compassion to often neglected disease. The following article was originally…
With all the political hocus-pocus that's enveloped the United States over the past year, I'm starting to get "petition burn-out," "marcher's burn-out," and just a general "call-to-action burn-out." This is…
I have a new hero, an Irishman who’s not only gotten some great press and notoriety, this guy has struck gold in the hearts of the Morquio syndrome community! In…
If you’re like most people (especially me), you’ve never heard of nicotinamide riboside (NR) before. But that may change very soon, especially for the Duchenne muscular dystrophy (DMD) community. What…
When I was 15 and received my learner's permit, they asked if I'd like to be an organ donor. I was a little freaked out at the image of getting…
Alveolar capillary dysplasia, or ACD, is an exceptionally rare disease that seldom has a positive outcome. It begins in the womb when the alveoli capillaries in baby's lungs don't properly…
I just had my yearly physical and something weird (for me) popped up in the blood tests. My cholesterol level was up. Not through the roof, but enough to raise…
Primary sclerosing cholangitis, or PSC, is a chronic progressive disease that damages the bile ducts. And, if you didn't know, bile is a liquid that assists the body in the…
What if you were born with an enzyme efficiency that prevented your body from breaking down lipids? Then you are probably one in 50,000 people who has Gaucher disease. And…
It is more blessed to give than to receive, and one man who lives with glomerulonephritis (GN) knows all too well how helping people and doing a good deed out…
Most people think of children when they hear cystic fibrosis. Thankfully with new treatments and new therapies people are living much longer. This gentleman, named Gary Anderson, is a perfect example.…
We can all say that our mothers gave us the gift of life. But how many can say that they gave the gift of life in return? One teenager in…
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