Life was so much simpler when we were kids. We didn’t have to worry about bills, or how what we say could accidentally offend someone else. Heck, even the stories…
According to the Rare Disease Report, 50 years ago, when Tommy Hoag was six years old, he received a kidney transplant. Tommy needed this transplant after having Scarlet fever and…
If you passed me as a stranger on the street, you would never guess that there was a point in time that I road tripped around the U.S. and picked…
Psoriasis is often labelled as “disgusting” or “contagious,” causing people who are afflicted by this disease to feel embarrassed and hide their skin from the rest of the world. But…
For a while, researchers and scientists have known that the protein, TDP-43, is found in clumps in the brain cells of patients with amyotrophic lateral sclerosis (ALS). It has also…
Being pregnant can cause some wonky things to happen to your body. Some go without saying—weight gain, strange cravings (jalapeño peppers on popcorn), fatigue (tbh, you’re carrying around another human…
Crinetics Pharmaceuticals, Inc. is a pharmaceutical/therapeutics company that focuses on rare endocrine-related disorders and cancers. This past month, excitingly, the pharmaceutical company announced that it had been awarded a Phase IIB…
Most people know the dangers of high blood pressure, high cholesterol, and high blood-sugar. But most people don’t think about high potassium as a problem. Unfortunately, high levels of potassium…
Cancer has a pretty complicated history with statins, a class of drugs that are most commonly used to lower blood cholesterol levels. In the past, laboratory tests involving statins have…
In 1998, The Mighty first brought the rare disease community into the spotlight, followed by Extraordinary Measures in 2010. Now, seven years later, The Big Sick is doing it again…
A breakout of pimples, a sunburn, and bad dandruff are all skin aliments that we probably faced while growing up, and all probably made us feel lousy, or at the…
Karl Swaine is nine years old. He has a rare progressive and genetic disorder called Hunter syndrome. This disorder prevents the body from breaking sugar molecules down. Throughout the world,…
CymaBay, Inc. recently announced positive provisional results from its low-dose Phase II seladelpar clinical trial for primary biliary cholangitis (PBC) patients. Once you get past the confusing part of that…
NeuroVia, Inc. is a rare disease pharmaceutical company that primarily focuses on finding treatments to rare neurological diseases. Markedly, this private company is especially concerned about the effects of the…
"Mom! What’s for dinner?" It’s a question that strikes fear in the hearts of matriarchs everywhere. Meats, breads, vegetables—you know the basics. Now add in a picky eater or someone…
Researchers have recently released some exciting news for Gaucher disease patients: a combination of two imaging techniques has been shown to better evaluate the risk of Gaucher patients developing bone…
When you think of an inspiring athlete, who comes to mind? Jesse Owens, who went to the 1936 Olympics held in Nazi-controlled Germany and proceeded to win four gold medals…
The pharmaceutical company, Kamada, has just submitted its proposed Phase III protocol for an inhaled alpha-1 antitrypsin therapy (inhaled AAT) to the U.S. Food and Drug Administration (FDA). This therapy…
The phrase “take advantage” is a bit strange. It can be quite innocuous with regards to using something. For example, there is nothing too bad about saying, “I’m going to…
As a teenager, my acne and periods were agonizing! I have high school yearbook pictures that still make me cringe. After gallons of over-the-counter acne medications and pain relief, it…
What happens when you imagine running a marathon? Unless you are an avid runner, the thought may make you laugh or even cringe. Running even one marathon may seem nearly…
It's getting hot out there... So you may as well read some news while staying cool inside if you're in the US! This week we have news on Senator John…
A new biotech company in Philadelphia called Enterin announced recently that their company has raised a whopping $12.7 million to support the hypothesis that Parkinson's disease starts in the gut,…
In early July of this year, the pharmaceutical company Bristol-Myers Squibb announced that the FDA approved of their new treatment, called abatacept (Orencia), either intravenously or subcutaneously. This drug is…
Earlier this month, Vertex Pharmaceuticals released big news regarding a new development in cystic fibrosis treatment. The pharmaceutical company announced that their new, innovative technique in treating the disease had…
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