According to a recent press release, biopharmaceutical company CASI Pharmaceuticals filed a Clinical Trial Application (CTA) (IND) in the United Kingdom. CASI Pharmaceuticals is hoping to test the safety…
International NASH Day, or IND, began in 2018 to raise awareness of nonalcoholic steatohepatitis (NASH). This is the most advanced form of fatty liver disease. It is a progressive condition…
According to a story from Scleroderma News, June is recognized as Scleroderma Awareness Month. While physical distancing as a result of the ongoing coronavirus/COVID-19 pandemic may keep some folks at…
According to a story from PR Newswire, Akcea Therapeutics, Inc. recently announced that the Portuguese Medicine Regulatory Authority (INFARMED) has announced its approval of reimbursement for inotersen (marketed as TEGSEDI®)…
Have you ever heard a sound that makes you want to clamp your hands over your ears? Well, for people with misophonia, this aversion to certain noises can be…
June is recognized as Myasthenia Gravis Awareness Month. Primarily organized by the Myasthenia Gravis Foundation of America (MGFA), this event is focused on spreading awareness as well as boosting efforts…
According to a story from PR Newswire, CureDuchenne Ventures has recently announced the initiation of its CureDuchenne Ventures 2020 Pitch Contest. This marks the first time that the contest has…
According to a story from BioSpace, the biopharmaceutical company MacroGenics, Inc. has just announced that the US Food and Drug Administration (FDA) has given Orphan Drug designation to the company's…
In a press release from Eli Lilly and Company, the company has announced recently that the US Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (BLA)…
According to a story from Multiple Sclerosis News Today, the drug company Bristol-Myers Squibb recently announced that its drug ozanimod (marketed as Zeposia) is now available for public use in…
According to Disability Horizons, an extremely talented filmmaker with muscular dystrophy recently won an Award of Recognition from The Best Shorts Film Competition. Stephanie Castelete-Tyrrell's film, entitled Muscle Warriors, documents…
According to a story from PR Newswire, the National Organization for Rare Disorders (NORD) has recently awarded a total of eleven new grant awards that will help fund essential research…
According to a story from EurekAlert!, a recent international study is utilizing data from 45 patients to describe a mostly unknown new rare genetic disorder. The researchers are recommending that…
Patient registries are a great way for patients to get involved in shaping future research around their conditions. Many times, getting involved in a rare disease (or rare disorder)…
According to a story from The Intercept, Gilead Sciences' drug remdesivir is being touted in the media as a major development in the treatment of COVID-19, the illness caused by…
According to a story from Sarcoidosis News, while the global challenge of the COVID-19 pandemic has been an obstacle for advancement in many rare diseases with trials and research grinding…
As reported in GSK, the FDA has just granted priority review to Nucala (mepolizumab), a targeted biological treatment to help patients with hypereosinophilic syndrome (HES). If the medication is accepted,…
Kimberly Underhay Kimberly Underhay is a Canadian who has survived cancer. She has also been diagnosed with osteoarthritis and has to use a walker. Her disabilities have left her unable…
Neurofibromatosis Neurofibromatosis is a rare condition that presents in different forms. Neurofibromatosis type one (NF1) causes tumors to grow on the plexiform neurofibromas, a type of nerve in the body. These tumors…
According to a story from globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US…
In high school, Chuck Graham loved sports. He was a student-athlete dedicated to his teams. But then, at 16 years old, a car accident paralyzed Graham from the chest…
According to Multiple Sclerosis News Today, the National Multiple Sclerosis Society recently released new exercise guidelines for people with multiple sclerosis. Exercise is extremely beneficial to health, for those…
According to a story from prnewswire.com, the Chiesi Global Rare Diseases and the biopharmaceutical company ProTalix Biotherapeutics have recently announced that they have submitted a Biologics License Application (BLA) for…
Christopher Cowan and his lab at the Medical University of South Carolina recently conducted a study in mice in order to understand how defective MEF2C genes impact brain development. A…
NORD has received thousands of phone calls and emails each week from concerned rare disease patients. They are asked for advice and how to decipher the myriad of contradictory information…
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