According to a story from BioSpace, Janssen Pharmaceutical Companies recently announced that the European Commission (EC) has expanded the approved indication of the company's drug ustekinumab (marketed as Stelara). This…
According to a story from newswise.com, the American Neuromuscular Foundation has recently awarded a grant to Dr. Karissa Gable. This grant will go towards a research project headed by Dr.…
According to a story from BioSpace, the genetic medicines company Passage Bio recently announced plans for its sixth program for gene therapy development. The company plans to work with the…
We're almost halfway through September believe it or not! As autumn approaches, we're continuing the aggregate rare disease news. This week, we're highlighting three studies, and one article on a…
According to a story from meaction.net, Jaime Seltzer, the Director of Scientific and Medical Outreach at #MEAction, recently spoke at the Third Annual Working Group Meeting on the Molecular Basis…
According to a story from firstworldpharma.com, the biopharmaceutical company Acceleron Pharma, Inc., recently announced that the company's experimental product candidate sotatercept has earned Orphan Drug designation from the US Food…
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The fountain of youth may actually be in a cocktail of drugs that were part of a new study on aging. A recent article in Longevity’s Science Research announced…
In the US, medical costs come in the form of drug prescriptions, doctors visits, expensive surgeries, and visits to specialists. As originally reported in The Tyee, in Canada, patients are…
According to a story from the Washington Post, the University of Virginia (UVA) Health System is often ranked as the best in the Old Dominion, but it also pursues debts…
According to a story from Hemophilia News Today, the drug developer uniQure has completed its goals for patient enrollment ahead of time for is phase 3 clinical trial. This clinical…
According to a story from BioPortfolio, Alexion Pharmaceuticals, Inc. and Eidos Therapeutics, Inc., have recently struck an agreement related to the development and marketing of an experimental treatment for hATTR…
According to a story from finanzen.at, the drug nintedanib (marketed as Ofev) has officially been approved by the US Food and Drug Administration (FDA) as a treatment for interstitial lung…
According to a story from EurekAlert!, farm workers from across the world are experiencing increased rates of chronic kidney disease. The precise reason for the increased rates are unknown, but…
According to a story from Buzzfeed News, Maria Isabel Bueso was born in Guatemala with mucopolysaccharidosis VI, also known as Maroteaux-Lamy syndrome. At age seven she traveled to the US…
September is Tyrosinemia Awareness Month! The good people of the Network Of Tyrosinemia Advocates (N.O.T.A.) have organized a family weekend later this month aimed to bring the Tyrosinemia community together. Let's…
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According to a recent article in Archive Today, an international band of amateur biologists, or "biohackers" announced that they intend to eventually market a knock-off of Glybera, a one…
According to a story from BioPortfolio, the drug company Alnylam Pharmaceuticals recently presented research findings related to hATTR amyloidosis, a rare disease. The study was presented at the Second European…
According to a story from The Globe and Mail, Canada's Patented Medicine Prices Review Board announced last month that drug prices would be subject to new regulations. These new rules…
Hey...Is That Autumn on the Horizon? Well...kind of but sort of no, it is still pretty hot where we are; but it is only a matter of time before we…
Maisie Green Maisie Green is a 20-month-old little girl whose insurance has finally approved coverage for a life-altering spinal muscular atrophy treatment. Two weeks ago she received the singular infusion…
According to a story from globenewswire.com, the biopharmaceutical company Processa Pharmaceuticals, Inc., has recently announced that it has completed enrollment for its phase 2 clinical trial. This trial will be…
According to a press release from California-based biotechnology company Zogenix, the Company recently completed a multi-million dollar acquisition of Modis Therapeutics. Before its acquisition, Modis focused on the development of…
According to a recent publication from Moneycontrol, patient advocacy groups in India are calling for the country's Minister of Health and Family Welfare, Harsh Vardhan, to establish a provisional system…
According to a publication from the Korea Times, the Eone Diagnomics Genome Center in Incheon, South Korea, is working to improve patients' accessibility to their own genetic sequence. Researchers believe…
According to a press release from American biotechnology company Fortress Biotech, the American Food and Drug Administration (FDA) recently awarded Regenerative Medicine Advanced Therapy designation to the experimental gene therapy…
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