Neuroblastoma, though rare, is the most common type of cancer in children who are five years old or younger. This cancer targets the immature nerve cells in the nerve tissue…
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!…
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:…
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…
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…
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…
Join Global Genes in honoring RARE champions of hope in the 2017 Tribute to Champions event in Anaheim, California! Hear from rare disease fighters and champions from all over the…
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…
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…
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…
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…
They say, “Necessity is the mother of invention.” This maxim goes for everything from nuclear technology (the Manhattan Project was a direct result of World War II) to the Snuggie®…
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