According to a story from BioPortfolio, the biopharmaceutical company X4 Pharmaceuticals, Inc. has recently initiated a phase 3 clinical trial that is testing its current lead product candidate mavorixafor. This…
According to a publication from ANCA Vasculitis News, a recent Spanish study of over 100 individuals diagnosed with ANCA-associated vasculitis found that major infections are a main cause of death…
According to a publication from the Warwick Beacon, friends and family of 4-year-old Banner Mazza are raising money for the boy's medical expenses. Mazza was recently diagnosed with ROHHAD, a…
According to a story from BioPortfolio, the drug company Pfizer has recently presented data from its phase 1b clinical trial that tested the company's experimental gene therapy PF-06939926, which is…
According to a press release from the Texas Biomedical Research Institute (Texas Biomed), researchers from the Institute have identified another new genetic mutation that they believe can cause rare congenital…
According to a story from The Boston Globe, the drug developer Ovid Therapeutics plans to move forward with a late stage clinical trial that will test the company's investigational drug…
Inflammatory Bowel Diseases Inflammatory bowel diseases (IBD) is the overarching term for both ulcerative colitis and Crohn's disease. In Canada, there are around 270,000 individuals living with the one of…
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The first indication of the influence that people can have in clinical trials when acting as a community may have occurred in the 1980s. As reported recently in Viral…
According to a press release from the Children's National Health System (a children's hospital in Washington, D.C.), two scientists from the Institution have been selected to receive federal funding grants…
According to a publication from EurekAlert, researchers from the University of Oklahoma College of Medicine (OUCM) recently published a study in the journal Gastroenterology suggesting how cachexia is triggered in…
According to a story from Find Me Cure, June is myasthenia gravis awareness month. Dr. Kemi Olawaiye-Dampson was first diagnosed with the disease after she gave birth to her first…
Ten-year-old Pennsylvania boy Josh Shorb has been hard at work raising money -- and awareness -- for a very rare disease he has, in an ultimate show of paying it…
According to a publication from the Arizona BioIndustry Association, Dr. Rachna Shroff and a team of researchers from the Arizona Cancer Center are studying the use of nab-paclitaxel (a pancreatic…
According to a story from Pharmaceutical Technology, the drug developer FibroGen's phase 3 clinical trial testing pamrevlumab as a treatment for idiopathic pulmonary fibrosis could be in trouble. While the…
According to a publication from the BBC, researchers with King's College London believe they've identified early biomarkers for Parkinson's disease that could help physicians predict who will be affected up…
According to a story from Newswise, scientists affiliated with the Huntsman Cancer Institute (HCI) of the University of Utah have found in recent lab studies that the experimental drug selinexor…
Sun's out, school's closed, summer's in full swing -- let's set aside some time to talk about juvenile idiopathic arthritis in the month of July! What is JIA? Juvenile idiopathic…
Researchers are studying gene replacement therapy in rare diseases at UT Southwestern Medical Center. Many rare diseases are caused from a defect in a single gene, so replacing the defective…
According to a story from metro.co.uk, many rare diseases and chronic conditions do not necessarily make a person look sick a lot of the time. This can make it harder…
According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…
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