According to a story from EurekAlert!, researchers have found that the drug Diamox, which is used to treat a variety of illnesses and conditions such as altitude sickness, glaucoma, epilepsy,…
According to a story from bioportfolio.com, two companies, Ionis Pharmaceuticals, Inc., and its affiliate Akcea Pharmaceuticals, recently announced that its product inotersen (to be marketed as TEGSEDI) has gained approval…
Catabasis Pharmaceuticals Inc. has announced plans for a Phase 3 clinical trial called Polaris DMD. It will investigate the experimental drug edasalonexent for the treatment of patients with Duchenne muscular…
According to a story from the Washington Times-Reporter, Michelle Marcinak's three sons-- John Henry, Leo, and Rafe-- were all diagnosed with the rare gastric condition eosinophilic esophagitis. Early symptoms of…
According to a story from BioSpace, the biopharmaceutical company Acceleron Pharma, which develops TGF-beta based treatments for rare and serious illnesses, recently announced that the U.S. Food and Drug Administration…
According to a story from ScienceDaily, a recent analysis has been conducted in order to investigate patient outcomes for children with the rare skin condition Stevens-Johnson syndrome, as well its…
The US Food and Drug Administration (FDA) has awarded Priority Review Designation to glasdegib, an experimental drug designed as a treatment for previously untreated acute myeloid leukaemia in combination with…
According to a story from NPR, plastic straw bans have been gaining traction across the U.S. One of the most shocking examples of planetary degradation as the result of human…
According to a story from the Globe and Mail, Michelle Quinlan was overwhelmed when her child Avery was diagnosed with Coffin-Siris syndrome, a rare genetic disease that can cause intellectual…
According to a story from Global News, direct-to-consumer genetic tests are becoming increasingly popular. These tests can be used to learn new info about your ancient ancestors, closer relatives, and,…
New pig models of Huntington’s disease have been developed, and they are expected to have several benefits compared to existing rodent models of the condition. Read the source article here…
Results from research into an experimental therapy called SL-401 for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm have been shared at the Congress of the European Haematology…
According to Medical Xpress, gene therapy may become the next treatment option for those with chronic kidney disease, CKD. Researchers at Washington University School of Medicine in St. Louis have…
Kalytera Therapeutics, Inc. has announced that a meeting with the Centre for Drug Evaluation and Research of the US FDA is planned for Tuesday, 31st July this year. The purpose of…
According to a story published by STAT, a daredevil neuroscientist at Stanford University is utilizing the relatively new concept of virtual reality to solve an unsuspecting problem. Not only is…
BrainStorm Cell Therapeutics Inc. has announced that it has secured its second U.S. manufacturing location for NurOwn®, an experimental drug currently under investigation for the treatment of amyotrophic lateral sclerosis…
According to a story from the Deccan Chronicle, researchers may have discovered a new treatment for Dupuytren's contracture that could offer substantial benefits over currently available treatment options. The scientists…
According to a story from medicalresearch.com, recent research has revealed that many pancreatic cancer tumors appear to have vulnerabilities to treatments that take advantage of genetic mutations that are present…
According to an article from the Detroit News, the most recent budget proposal from the Trump administration would call for cuts to Medicare to the tune of $236 billion over…
Bruce Howell is 63 years old living with retinitis pigmentosa. He’s been legally blind since age 37. As a result, public transportation is a critical service to him. Without the…
According to a story from pm360online.com, a new study of Prader-Willi syndrome will soon be under way thanks to a recent collaboration between the biopharmaceutical company Zafgen and the Foundation…
According to a story from Psychology Today, a study from 2017 titled Cognitive behavioral therapy is effective in misophonia: An open trial concluded that cognitive behavioral therapy could play a significant role in…
Ellil Mathiyan is the sort of lively character most people can’t believe experienced a double cancer diagnosis. He describes how people usually respond with “are you sure?” or “you’re kidding”…
As I’ve gotten older, I realize I’ve become more introspective, analyzing my relationships, emotions, spiritual well being, and mental health. While talking to others with rare diseases I’ve learned it…
Organisations are planning to develop new clinical practice guidelines for the diagnosis and management of von Willebrand disease. The original article can be read here, at PR Newswire (whose source…
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