In very recent news, the FDA's Arthritis Advisory Committee convened to evaluate a new drug, and excitingly, voted in favor of it. The drug is question was tofacitinib citrate (Xeljanz) for…
Imagine if every time you stood up your world would start spinning, your vision would blur, and you’d faint or fall down. Prolonged exercise is out of the question. Extreme…
Organ donation is an intensive process to go through. It takes a ton of time to find a match, and even after the donation has taken place, recipients run a…
Soleno Therapeutics, Inc. of Capnia reported in early July of this year that the company plans to start a Phase 3 clinical trial for evaluating diazoxide choline controlled-release (DCCR) in…
The latest in Waldenstrom macroglobulinemia news comes not only from the hospital clinic, but also from the race track, and the dance floor! As for the former, latest results are…
Happy August Patient Worthy Peeps! This week, we have some awesome posts to keep you up-to-date on some of the latest rare disease developments. There's news on a hopeful possible…
Medical malpractice. These two words strike fear into the hearts of millions of doctors all over the world, but especially in litigious societies, such as the United States. Not all…
Recently published findings from the Journal of Nuclear Cardiology reported that a novel imaging method is much more accurate and thorough in diagnosing cardiac sarcoidosis than traditional ones. Sarcoidosis is a rare…
Major breakthrough alert! Scientists have developed a method to test the sweat of infants to detect cystic fibrosis (CF). CF is an inherited disorder that causes severe damage to the…
This is not a warning, but some friendly advice: Start planning for next year and mark your calendar for February 28, 2018—it’s world Rare Disease Day! It’s the day that…
In late July of this year, biopharmaceutical company, Incyte, announced that the very first treatment in a Phase 3 clinical trial for itacitinib had been administered for an acute graft-versus-host…
I wouldn’t exactly call myself a meditation guru. I was first introduced to the concept of meditation after a class on Buddhism in college. I attempted to mediate for several…
If you’re a science nerd, we live in some pretty exciting times. Even though we’re going through a moment where it feels like science and empirical evidence itself is under…
Constipation. It's not the first symptom you think of when considering the effects of Parkinson's Disease (PD). Tremors, rigidity, insomnia, and depression are all well-known symptoms of PD. Constipation? Not…
Have you ever noticed how frequently things get compared to the same small group of ideas? When trying to point out that something isn’t as complex as it appears, you…
As rare, neurodegenerative diseases go, amyotrophic lateral sclerosis—or ALS—“enjoys” an unusually high profile. This is due in no small part to both the ice bucket challenge that was everywhere a…
Although there are treatments currently available with a proven track record against rare types of cancers, such as Waldenstrom macroglobulinemia, with any treatment, however successful, there will be a handful…
It's hard to believe it's already been about a month since the hemophilia A community received this exciting news! In case you missed it, there's a new drug that got…
Genetic engineering has, for a long time, been a highly heated and contested issue. One must weigh the ethical ramifications and risks of engineering human embryos with the huge potential…
While multiple myeloma treatment has made significant advances in recent years, the prospect of a cure remains as elusive as ever—just as it does for all cancers. But that may…
A little thing called "genomic technology" could just be a game changer for people who suffer from idiopathic pulmonary fibrosis (IPF.) What is genomic technology? Glad you asked. Genomics is…
In the mid-1990s, many in the United States were first exposed to the somewhat strange juxtaposition of choreographed leg flailing and still upper bodies. This was, of course, Riverdance. This…
At some point in the past, we decided that it would be important to divide things up when we study them in school. For a long time, all science was…
We’ve all been there. In the grocery store or at the mall or at the amusement park, when a child starts to act out, much to the chagrin of the…
There’s a lot that the marginal zone lymphoma community needs, but there is also a lot being said. Results from clinical trials are coming in, and patient testimonials are shedding…
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