According to a story from mdmag.com, a phase 2 trial testing the drug avapritinib as a treatment for systemic mastocytosis is currently ongoing. The results so far suggest that the…
According to a story from the National Gaucher Foundation, the use of gene therapy in order to treat Gaucher disease has long been floated in medical theory, but only in…
According to a story from Benzinga, the Novartis Company AveXis recently announced the results of long term studies testing the impact of Zolgensma, a gene therapy treatment that was approved…
According to a story from The Intercept, Gilead Sciences' antiviral drug remdesivir, which is being tested as a treatment for coronavirus/COVID-19, was just given Orphan Drug designation by the US…
According to a story from Healio, a recent study that was published in the scientific journal BMC Rheumatology revealed that it was uncommon for patients living with ankylosing spondylitis to switch from…
The American Kidney Fund (AKF) has recently announced the formation of a Coronavirus Emergency Fund intended to support low income dialysis and transplant patients who may face unanticipated financial challenges…
According to a story from Healio, chronic kidney disease and diabetes are closely related. Kidney disease is unfortunately a frequent complication of both type 1 and 2 diabetes. In the…
According to a story from gurufocus, the biotechnology company QED Therapeutics recently announced that the first patients have been dosed in separate phase 2 and phase 3 clinical studies testing…
According to a story from The Scarborough News, brother Connor and sister Kiera Pickering, aged eleven and twelve years respectively, will be some of the first patients to be treated…
According to a story from NeurologyLive, patients being treated for autoimmune diseases such as multiple sclerosis (MS) or myasthenia gravis could be at heightened risk of infection with coronavirus because…
According to a story from BioSpace, GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical, Inc. have recently announced that the first patient has been dosed in the companies' phase 1/2 clinical trial.…
According to a story from BioSpace, the testing of drugs in clinical trials to address the current coronavirus/COVID-19 pandemic will soon be under way. The biopharmaceutical company Sanofi and the…
According to a story from Independent, a team of scientists from Canada have isolated the strain of coronavirus that has rapidly spread around the globe in recent months. What this…
According to a story from Yahoo News, Dr. Nancy Wexler spent around 20 years in Venezuela conducting pivotal research on a deadly rare genetic disorder: Huntington's disease. On Lake Maracaibo,…
According to a story from LocalHealthGuide, COVID-19 has continued to spread through the US and the lackluster government response suggests that it isn't going to be contained any time soon.…
According to a story from Hindawi, a recent study tested a combination treatment for patients with metastatic or locally advanced soft tissue sarcoma. Outcomes for these patients are poor if…
According to a story from Healio, Dr. Adam Cheifetz recently spoke about treatment for inflammatory bowel disease (IBD) at GUILD Conference 2020. He says that it is time for each…
According to a story from Healio, researchers are hard at work determining who could be at the greatest risk from infection with COVID-19/coronavirus. So far, the elderly, people with heart…
Antonio Maltese is a young man living in Virginia. His family has also been devastated by the hereditary rare illness called Huntington's disease. In fact, he was diagnosed with the…
According to a story from The Charlotte Observer, Chris Younger was 18 when he first was diagnosed with X-linked hypophosphatemia, a rare disease that affects the bones. However, he had…
According to a story from Yahoo Finance, the biopharmaceutical company Mallinckrodt recently released findings from an open label pilot study which tested the impact of Acthar Gel (corticotropin injection) as…
According to a story from Charcot-Marie-Tooth News, a new study highlights the advances that have been made in the last decade for treating rare diseases that affect children. However, the…
According to a story from businesswire.com, the Bristol Myers Squibb Company recently released the topline results from its phase 3 clinical trial. This study was testing a combination treatment consisting…
According to a story from Healing Hugs Haven, the rapid spread of COVID-19 around the world has been getting a lot of airtime in the news lately, and for good…
According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…
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