According to a press release from American biotechnology company Intercept Pharmaceuticals, the company recently released additional data supporting its phase 3 clinical trial of obeticholic acid (OCA) in the treatment…
Six weeks ago, Gretchen and Josh of Oklahoma rushed their precious 5-month-old Opal to the hospital after she became extremely sick from a cold. It didn't take doctors too long…
April 15th is International Pompe Disease Day! The International Pompe Association (IPA) is an organization of Pompe disease patient groups that seeks to coordinate activities and share experiences worldwide. The…
According to a story from Rocket News, the class of drugs known for their "gene silencing" mechanism of action appear to be effective in treating porphyria, a rare disease that…
It's one thing to cope with a new rare disease diagnosis. It's another entirely to cope with that diagnosis and then four years later learn that diagnosis is incorrect and…
Biophosphonates (BPs) are common treatments for osteoporosis and metastatic bone cancer. They help to prevent bone loss. Unfortunately, these drugs can have serious side effects. One of the most severe…
Danon Disease Danon disease (DD) is an extremely rare condition that is not very well understood in the medical community. It was first described in 1981, and it is named…
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
According to a story from CRWE World, the genetic medicines company Audentes Therapeutics has recently announced its intent to develop new therapies for myotonic dystrophy and Duchenne muscular dystrophy. These…
According to a press release from Inovio Pharmaceuticals, the American biotech company's experimental recurrent respiratory papillomatosis (RRP) drug INO-3106 showed promising results at the end of a pilot clinical study…
According to a story from finanzen.at, the biotechnology company Orbsen Therapeutics recently announced that its immunotherapy product ORBCEL-C is to be tested in a multi-site clinical trial called MERLIN that…
People living with rare diseases such as amyotrophic lateral sclerosis (ALS) or muscular dystrophy as well as those who have suffered from a stroke or spinal cord injury often experience…
According to a story from Xconomy, a recent report from the Institute for Clinical and Economic Review (ICER), based on Boston, is calling for reductions to the price tags of…
According to a publication from Multiple Sclerosis News Today, recent studies of mesenchymal stem cells (MSCs - adult stem cells) suggest that autologous transplant of stem cells could one day…
According to a recent article in Check Orphan, genetic and biochemical testing are combined in a new method of metabolic testing called the CentoMetabolic panel. The announcement comes from…
According to a story from Medical Xpress, a recent study conducted by researchers from the UNC School of Medicine has illustrated that the accumulation of sticky, abnormal mucus, along with…
Mastocytosis Mastocytosis is a rare disease caused by mutations in the KIT gene. The condition causes mast cells to accumulate in the body. Depending on the location of these immune…
What Are The Differences and Similarities Between MS and ALS? There are some similarities between the two. For example, both diseases are neurodegenerative. This means that both diseases damage…
According to a publication from health website Stat, a new way of thinking about the price of drugs is taking root in Washington D.C. and beyond. "Value-based agreements" are a…
According to a publication from Neuroscience News, a team of researchers has managed to examine how amyotrophic lateral sclerosis (ALS) progresses on a cellular level for the first time. The…
According to a story from Cystic Fibrosis News Today, Elizabeth Rogers, a cystic fibrosis patient, is just one of the many who have been disappointed by the lack of impact…
In a recent video interview with Healio, Dr. David Rubin recapped some of his discussion at the 2019 Interdisciplinary Autoimmune Summit. At the summit, he and his colleagues spoke about…
According to a recent article from CBC News, two patients treated for cataracts at Moncton Hospital in New Brunswick this winter were later diagnosed with Creutzfeldt-Jakob disease. The prion disease…
According to a press release from American biotechnology company Soligenix, the European Medicines Agency's (EMA) Pediatric Committee recently agreed to the Company’s Pediatric Investigation Plan (PIP) for experimental oral mucositis…
According to a press release from the Muscular Dystrophy Association published at Biospace, the group has resolved to award eight new research grants for the study of amyotrophic lateral sclerosis…
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