Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…
Genetic Counselors are an incredible resource – they can help assess your risk of developing a condition, the risk of your children inheriting a condition, and what potential treatment or…
“I feel like I’m in a huge dream that nobody can get into,” Noemie explained, describing her weeks of intensive sleep during which she slept 20 hours a day, the…
Sometimes in moments of tragedy, there is a bright silver lining that helps us learn, and that's exactly what Amber Briggle was aiming to do. Back in October, Amber posted…
No healthcare professional wants to hear that a drug being studied in a clinical trial did not meet the endpoints. It means that after countless hours of hard work,…
It is estimated that about 10% of people in the United States have rare diseases. But what makes a disease rare? How does this distinction affect diagnosis and treatment?…
As originally reported in MSU Today; in new experiments on mice, researchers looked into how the TS gene is related to autism. Tuberous sclerosis is a rare disorder known for…
As originally reported in the Hindu Times; getting healthcare for a rare disease is hard, in general. 95% of rare disease patients have no therapy options available. Thats 380 million…
In the world of rare diseases, progress can often feel like a slow and agonizing slog. In many cases, this is the unfortunate reality. However, a lot can change in…
As originally reported in Pulmonary Hypertension News, sleep is a natural cleanser for the body. During the nights, our brain imbeds our memories, removes metabolic waste, and heals itself and…
As originally reported in the Cambridge Independent; rare disease treatment is full of potential. In part, because there aren’t many. This is not for lack of ability, but for lack…
As originally reported in Medscape; many rare disease patients know the frustration of not having any drugs or treatments available for their disease. 95% of rare disease patients have no…
If you are unable to work because of a medical condition, you may qualify for disability benefits from the Social Security Administration (SSA). The SSA oversees two different disability programs, Social Security Disability…
According to a story from BioPortfolio, the drug companies bluebird bio, Inc. and the Bristol-Myers Squibb Company have recently announced positive final results for their phase 2 clinical trial. This…
According to a story from globenewswire.com, the biopharmaceutical company Lexicon Pharmaceuticals has recently announced that it has completed its safety review of the initial cohort for its phase 2 clinical…
According to a story from mHealth Intelligence, the University of Alabama has found success with using telehealth to check in with kidney disease patients that are on dialysis. Now, it…
A drug created by Agios Pharmaceuticals, TIBSOVO, was recently granted the Breakthrough Therapy designation by the FDA for the treatment of relapsed or refractory myelodysplastic syndrome (MDS) with an…
According to a story from Pulmonary Hypertension News, a recent study has determined that cardiopulmonary exercise testing can be an effective method to help identify systemic sclerosis patients that are…
Happy Holidays, Zebra Community! As we gear up for the holiday season, we're highlighting four articles about managing rare disease during the holidays. We have some new articles about spending…
According to a story from Medscape, an advisory panel from the US Food and Drug Administration (FDA) has recommended the approval of the drug teprotumumab for intravenous administration as a…
Mantle cell lymphoma (MCL) is a very rare form of cancer, and oncologists may only see a few cases of it through the course of their careers. Because of…
According to a story from PR Newswire, the biopharmaceutical company Soligenix, Inc. has announced recently that its phase 3 clinical trial has officially completed enrollment. This trial is testing the…
According to a story from the Star Tribune, being disabled in Minnesota can be a damaging affair. The state's most common method for caring for disabled people are group homes,…
According to a story from prnewswire.com, the biopharmaceutical company InMed Pharmaceuticals, Inc. has recently announced that that company's Clinical Trial Application has been approved in the Netherlands. This approval will…
There is no known cure for sickle cell disease. Treatment is symptomatic and meant to prolong life. While a blood and bone marrow transplant is an option, not everyone…
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