There's a lot happening in the cystic fibrosis world, so here's the latest scoop. Cystic fibrosis (CF) is a genetic disease that causes progressive and constant lung infections as well as…
A recent study found that a specific bacteria may be the distinguishing factor between cardiac sarcoidosis and other conditions that affect the heart. This data suggests that this bacteria may…
There’s a lot happening in the adrenoleukodystrophy (ALD) world, so here’s the latest scoop. Adrenoleukodystrophy, or ALD for short, is a genetic rare disease that destroys the protective sheath surrounding…
Recently, the U.S. FDA has deemed a particular idiopathic pulmonary fibrosis (IPF) drug "orphan drug status" worthy. Last week, on July 25, 2017, it was reported that the FDA granted…
Scientists may have discovered something crucial for the future of Parkinson's research. They found a new way to observe changes in the brain from Parkinson's disease. Specifically, research suggests that…
A report titled, “Association of Sarcoidosis With Increased Risk of VTE: A Population-Based Study, 1976 to 2013,” recently released information that patients with sarcoidosis have a 3-fold increased risk for…
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…
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…
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…
July is the month of a lot of great things: The Fourth of July, long summer nights, fresh fruit, fireflies, and summer vacation. But there's something else great about this…
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…
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…
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…
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…
The United Leukodystrophy Foundation is hosting two two-day long events for families and medical professionals! Here's all you need to know about it before registering: What: Cocktail reception and dinner, fascinating…
Two lucky Lennox-Gastaut syndrome (LGS) researchers were awarded grants by the LGS Foundation last week on July 12. These two recipients are both doing fascinating work in LGS research and,…
Have you ever been handed a questionnaire at the doctor's office and wondered why and how it was relevant? Where does it go? Who has the time to read all…
If you're a millennial, or if you are close to a millennial, or actually if you just have a social media presence at all, you have probably heard of the…
Researchers have just found some really exciting information that will be crucial in the development of idiopathic pulmonary fibrosis (IPF) treatments from here on out. Scientists at Northwestern Medicine have…
There's some new information available about Parkinson's disease, and researchers are really excited about it. Earlier this month, researchers found that visual system changes in recently diagnosed Parkinson's patients can…
Bleeding is painful. It's not only painful, but probably worse, it's also just a nuisance and a hassle. Lucky for most of us, pressure, a bandaid, or, at worst, a…
On May 23, 2017, a biopharmaceutical company, ChemoCentryx, Inc., made a huge announcement. Not only has the US Food and Drug Administration (FDA) granted orphan drug designation for their drug, avacopan,…
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