PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…
According to a story from med.miami.edu, a team of researchers working at the Sylvester Comprehensive Cancer Center, which is part of the University of Miami School of Medicine, made a…
According to a story from the National Institutes of Health, a recent study has revealed that the absence of a specific protein in the cells lining the esophagus could be…
According to a story from PLOS Medicine, most people will probably tell you that the anti-vaccine movement in the United States is a total failure. While the vast majority of…
According to a story from Rare Disease Report, Nichol Kirby, who was diagnosed with acute intermittent porphyria in 2015, faced an arduous path to her diagnosis. Her constant pain, which…
According to a story from Mirror, the advocacy group Patient Concern is calling out the UK's National Health Service (NHS) for failing to treat cancer patients appropriately. The claims are…
According to a story from abc.net.au, ever since Massimo Damiani was first diagnosed with a leukodystrophy, his parents, Sally and Stephen, have been committed to promoting research efforts and new…
According to a story from pm360online.com, the clinical stage corporation Epizyme recently released detailed results from the Phase 2 trial of its leading investigational candidate, tazemetostat. This drug is in…
According to a story from msn.com, a significant portion of woman may notice a small number of hairs growing on their chin at some point in their lives. In most…
According to a story from empr.com, a recent study revealed that gene therapy could allow patients with beta thalessemia to stop having to get regular blood transfusions. The gene therapy…
According to a story from Reuters, the cancer drug developer Loxo Oncology recently released the latest results from the trial testing its investigational therapy LOXO-292. Preliminary results released about a…
According to a story from The Guardian, doctors are celebrating the effectiveness of an all new, experimental immunotherapy that was able to totally eliminate a case of metastatic breast cancer…
According to a story from the Pharma Letter, the Indian government has decided to take some steps to make rare disease and orphan drugs more affordable for its citizens. The…
According to a story from PR Newswire, the U.S. Food and Drug Administration (FDA) recently approved the expansion of the indication for tofacitinib (marketed as Xeljanz), which will now include treatment…
According to a story from Renal & Urology News, a recent study investigated the origins of hyperkalemia caused by certain treatments, specifically the class of drugs known as renin-angiotensin-aldosterone system inhibitors…
According to a story from cancer.nautil.us, some cancer researchers are beginning to look at the animal kingdom in order to find new answers for finding what it takes to beat…
According to a story from Reuters, the Danish biotech company Genmab saw its share value drop by a quarter after Johnson & Johnson, which had partnered with the company to…
According to a story from the Oxford Mail, Stuart and Kira Connor were devastated when their daughter Grace, who was only three years old, suddenly passed away because of mitochondrial…
Myalgic encephalomyelitis/ chronic fatigue syndrome (ME/CFS) has been a controversial condition ever since it was first diagnosed and recognized. Some uninformed doctors still believe that the syndrome is a psychological…
According to a story from apnews.com, President Trump signed the "Right to Try" bill passed by Congress into law on Wednesday, May 30th, 2018. During the signing ceremony, he was…
According to a story from the Daily Herald, Kevin Barnes, a man from Wauconda, Illinois, believes that his experience with esophageal cancer helped spur his own spiritual growth. This is…
According to an article from Globe Newswire, the company Shire plc recently presented new data in relation to recombinant human parathyroid hormone (rhPTH) and its relationship to hypoparathyroidism. This information…
After years of resistance from the previously dominant Republican party, Virginia lawmakers were successfully able to pass a measure expanding Medicaid to nearly 400,000 more Virginians, according to the Washington…
According to a story from Business Wire, the company CENTOGENE, which is dedicated to researching rare genetic disease, will be partnering with the biotech company Aldeyra Therapeutics, Inc. This partnership…
According to a story from pm360online.com, the U.S. Food and Drug Administration (FDA), has given Priority Review status to eltrombopag (marketed as Promacta) after impressive clinical trial results. Eltrombopag was…
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