An Idea Sometimes life leads us down unexpected paths. Mark Greget founded a medical device distribution company. That was his plan. But after interacting with patients through his work, he…
Happy Thursday! Today, we're highlighting a piece from a PW contributor sharing why it's important for people in the rare disease community to vent sometimes. If you'd like to use…
According to a story from BioPortfolio, the gene therapy company bluebird bio, Inc. recently announced that its gene therapy ZYNTEGLO™ has earned conditional marketing authorization in the European Union from…
According to a story from BioSpace, the drug developer Daiichi Sankyo Company Limited has recently announced positive data resulting from a pooled anaylsis of a phase 3 trial and a…
The Problem One in every 12 Canadians will be diagnosed with a rare disease in their lifetime. Two/thirds of these individuals are children and most of these patients experience debilitating…
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Ron Davis and his gene-sequencing technologies were mentioned years ago in The Atlantic alongside Elon Musk (SpaceX) and Jeff Bezos of Amazon fame. Since then, Davis has amassed over…
According to a story from statnews.com, one pharmaceutical company is taking advantage of a little known loophole in the Orphan Drug Act to suppress competition and rake in gluttonous profits.…
According to a story from Science Daily, a team of scientists from Johns Hopkins Medicine conducted a review of studies going back nearly 50 years and have concluded that surgery…
In a letter to Dr. Norman Sharpless, the acting commissioner of the Food and Drug Administration since April 5, Senator Ted Cruz encouraged the federal agency to expand the number…
We all run into problems in our everyday lives that cause frustrations and test our patience. If we're feeling well, we react to those issues at the moment and then…
According to a story from the South China Morning Post, there are an estimated 20 million people in China that are rare disease patients. The Chinese government has 121 diseases…
According to a story from the website of Oregon rep Earl Blumenauer, Blumenauer (D-OR) along with fellow congressmen George Holding (R-NC) and G. K. Butterfield (D-NC) have introduced a bill…
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…
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