According to a story from Hospital Healthcare Europe, The European Medicines Agency approved Kineret last year, an anti-inflammatory drug developed by Sobi. In the UK, Kineret is an approved treatment…
According to a story reported by Global News Canada, Michelle Quinlan said it was seven years before doctors correctly diagnosed her daughter, Avery, with Coffin-Siris syndrome. A new project by…
UPDATE: Patisiran has been approved! Check out the story here. According to a story from Xconomy, the U.S. Food and Drug Administration could be close to approving a new drug…
According to a source article on Business Wire that can be found here, the Committee for Orphan Medicinal Products (part of the European Medicines Agency) has recommended that the experimental…
According to a report by EuroInvestor, biopharmaceutical company Novelion Therapeutics, Inc. was recently granted permission to market their drug MYALEPTA. Novelion intends MYALEPTA to function as a method of treating…
According to a story from businesswire.com, Otsuka Pharmaceutical Europe Ltd recently announced that its product JINARC, also known as tolvaptan, now has an expanded therapeutic indication after a recent approval…
Patient Worthy exists because there's just not enough visibility for the nearly 30 million Americans who have been diagnosed with disorders that are rare or have limited treatment options. So…
According to a story from BioSpace, an announcement from the biotechnology company Vertex Pharmaceuticals revealed that the U.S. Food and Drug Administration has approved an expanded indication for ORKAMBI, the…
According to a story by Mirror, the Rare Disease Centre at Birmingham Children’s Hospital is set to begin operations after a massive donation by readers of The Star Appeal. Readers…
According to a story from Benzinga, the pharmaceutical company BioCryst Pharmaceuticals, Inc., recently announced that its investigational product BCX7353 has been granted Fast Track Designation from the FDA for the…
These groundbreaking research results have been published on the website of the University in Erlangen and in the New England Journal of Medicine, and I would like to share them…
According to a story from MPR News, Rae Baylark, who is the founder and current president of the Sickle Cell Foundation of Minnesota, felt like she had nowhere to seek…
According to a story from the pharmaceutical company Takeda, the company recently released the results from its clinical trial testing an experimental subcutaneous formula of the drug vedolizumab. The drug…
According to a story from Beyond the Dish, a new experimental treatment for spinocerebellar ataxia was given the go-ahead from the U.S. Food and Drug Administration for testing. The investigational…
According to a source article that can be found here on Ultragenyx’s website, the first patient has been dosed in a trial of an investigational gene therapy called DTX401 that…
According to University of Wisconsin News, UW-Madison professor David Beebe and his colleague Anna Huttenlocher, fellow UW professor, were awarded a $3.7 million grant from the National Institutes of Health (NIH)…
According to a story from PR Newswire, the U.S. Food and Drug Administration had approved the New Drug Application for an innovative new delivery system for Remodulin, a therapy that…
According to Business Wire, a pharmaceutical company, Aurinia Pharmaceuticals Inc., recently announced that they have enrolled the first lupus nephritis patients in the AURORA 2 extension study. Not only this, but Aurinia…
According to UPI, a recent study has found that standard Parkinson's disease treatments may be responsible for a plethora of other things as well. Most notably, those who take…
According to a story from the FH Foundation, the non-profit organization has released a vital consensus statement regarding the diagnosis of familial hypercholesterolemia, which is the most prevalent genetic cause…
According to a story from pcmag.com, the pharmaceutical company GlaxoSmithKline (GSK) has recently partnered with 23andMe as part of a four year long agreement that will grant the company exclusive…
According to a story from globenewswire.com, the company Progenic Pharmaceuticals recently reported the the U.S. Food and Drug Administration has recently approved the company's product iobenguane I 131 (AZEDRA). This…
What do Dave Winfield, Steve Garvey, and Don Baylor Jr. all have in common? Besides all being big names in major league baseball, each of the famous athletes is contributing…
Happy End-of-July! As the week comes to a close, we want to spotlight four articles. We have an honest piece from PW contributor, Tom Seaman. Next, we investigate the root…
According to a story from Business Wire, the non-profit foundation CurePSP has agreed to allocate funding for four venture grants. The funding is worth over $300,000 and will be directed…
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