For those who haven't finished watching HBO's Game of Thrones, this article contains spoilers for the series finale! If you don't want the ending ruined, read no further! Well, that…
Happy Thursday! Today, we're highlighting two patient stories on adjusting to life and staying strong after a rare diagnosis. Next, we have an article about parents of children with Batten…
The FDA has just approved the very first therapy for malignant pleural mesothelioma (MPM) in more than 15 years. It is the NovoTTF-100L System, developed by Novocure. Not only is…
Living with a chronic long-term health condition, can have a devastating effect on someone’s life as it affects everything we do. It’s like finding yourself on a new path without…
According to a story from globenewswire.com, the biopharmaceutical company Cellectar Biosciences, Inc. has announced plans to expand the third treatment cohort in its ongoing phase 2 clinical trial. This clinical…
The Cure Rare Disease Foundation The Cure Rare Disease Foundation's mission is to increase collaborative efforts between researchers in order to accelerate the development of customized therapies for individuals living with…
According to a story from Science Daily, a review of studies focused on allogeneic stem cell transplants as a treatment for non-Hodgkin lymphoma found that it wasn't possible to conclude…
According to a story from PR Newswire, the Novartis company AveXis recently announced that the US Food and Drug Administration (FDA) has approved Zolgensma, making it the first ever gene…
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Newly diagnosed patients may decide to either receive medical treatment or forego treatment based on the results of their diagnostic test. As recently reported in PEW, although it is…
Claudia Stäubert and her team of researchers at the University of Leipzig have just reported noteworthy findings which could impact the future treatment of inflammatory diseases. Lactic acid bacteria, or…
June is myasthenia gravis awareness month! Myasthenia gravis (MG) is characterized by weakness and rapid fatigue of any of the muscles under voluntary control. It's caused by a breakdown in…
According to a story from apnews.com, the gene and cell therapy company Abeona Therapeutics, Inc., has recently announced that the US Food and Drug Administration (FDA) has given the company's…
According to a story from the National Gaucher Foundation, the stress and difficulties of living with a rare or chronic disease can take a major toll on a patient's mental…
The International Waldenstrom's Macroglobulinemia Foundation (IWMF) is a nonprofit organization founded by patients. Their mission? Support patients currently living with Waldenstrom's Macroglobulinemia (WM) while simultaneously supporting the search for a…
According to a story from Financial Buzz, the drug developer Rafael Pharmaceuticals, Inc., recently announced that a Phase II clinical trial that will test the company's experimental drug CPI-613 in…
Happy Thursday! We hope everyone's enjoying the spring weather. Today, we're highlighting a story from a young man advocating for Huntington's patients. Next, we have an article about a surprising…
My name is Antonio Maltese and I am a 22 year old senior Political Science major concentrating in International Relations and minoring in German at Virginia Commonwealth University. Back in…
According to a story from MD Magazine, a recent study that was presented at Digestive Disease Week 2019 suggests that patients with ulcerative colitis, a rare form of inflammatory bowel…
Q&A with NORD Director of Membership, Debbie Drell, on the Living Rare, Living Stronger Patient and Family Forum, June 21-23 in Houston, Texas Which health care professionals would most benefit…
According to a story from The Washington Post, a host of different factors ultimately led to the tragic death of Olivia Shea Paregol, a freshman at the University of Maryland.…
According to a publication from Express Digest, Britain's National Health Service (NHS) has finally reached an agreement with American biotechnology company Biogen Inc. over the price of Spinraza - a…
According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company's investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US…
According to a story from Kamloops Coffee Talk, Darrin Rein, of Kamloops, British Columbia, was just recently diagnosed with spinocerebellar ataxia, a rare disease that affects mobility and coordination. Despite…
Short Bowel Syndrome Short bowel syndrome (SBS) is a rare condition which occurs when patients have a large portion of their intestine removed. The surgery which leads to this diagnosis…
The Word "Rare" Unfortunately, many experts in rare disease believe the phrase "rare disease" may be harming the very population it describes. The Director of the National Center for Advancing…
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