Tissue engineering has a relatively easy concept behind it. First, a patient’s stem cells are grown in the laboratory. Then, they are added to a scaffold material. And ta da!…
Jon Milne is on a quest to race across the seven seas to help raise awareness of rare disease! Jon has always been a keen sportsman, aged 30 he had…
Global Genes is hosting its 6th Annual RARE Patient Advocacy Summit for the rare disease community! Here's all you need to know about it, and why you won't want to miss it:…
It started out harmlessly enough—a Greatest Loser- type competition between co-workers. Everyone who wanted to participate put in $10, weigh-ins would be weekly, and the person who lost the most…
Don’t eat that! Eat this instead. Some diets recommend low fat. Some endorse high protein. Some forbid carbohydrates. Some praise those same carbohydrates. Some limit fiber. It’s so confusing figuring…
A new therapy option for hereditary angioedema (HAE) has recently received FDA approval! Marketed as Haegarda by biopharmaceutical company, CSL Behring, this subcutaneous C1 Esterase Inhibitor is now usable in…
BioMarin just announced interim results from a continuing Phase 1/2 study for hemophilia A patients, testing a gene therapy known as BMN 270. Hemophilia A is a rare bleeding disease…
The other day while I was shadowing a neurologist, I encountered a multiple sclerosis (MS) patient. The patient's MS was stable and he was doing quite well on his medications.…
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…
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