According to a story from BioSpace, the pharmaceutical company Marinus Pharmaceuticals, Inc. announced recently that its investigational product candidate ganaxolone has earned Rare Pediatric Disease designation from the US Food…
The HEALY ALS Platform Trial, being conducted by Biohaven Pharma, has begun enrolling patients. This study is intended to test the efficacy of three treatments on amyotrophic lateral sclerosis (ALS),…
CymaBay Therapeutics has recently released the results from ENHANCE, a study of seladelpar as a treatment for primary biliary cholangitis (PBC). These results were positive, proving that this medication has…
Alnylam Pharmaceuticals has submitted their clinical trial authorization (CTA) application to the Medicines and Healthcare Products Regulatory Agency (MHRA) for their RNAi therapy, ALN-HSD, a treatment for nonalcoholic steatohepatitis (NASH).…
According to Ryan McDonald of Cure Today, the FDA recently granted Breakthrough Therapy Designation to MK-6482, an investigational HIF-2α inhibitor developed by Merck. The orally-administered drug, designed to treat…
Within the last week, biotechnology company Levo Therapeutics announced results from the Phase 3 CARE-PWS clinical trial. During the trial, researchers examined the efficacy, safety, and tolerability of LV-101…
Rare disease or health-related foundations often encourage patient advocacy, research, and discussion around new treatments. According to Parkinson's News Today, the Parkinson's Foundation is doing their part in advancing…
Earlier this week, biotechnology company Time Technologies announced that its drug candidate SM-88 received Orphan Drug Designation from the FDA for the treatment of pancreatic cancer. The FDA defines…
Living with a rare disease can be difficult for a myriad of reasons, one of them being financial. Treatments, appointments, tests, and everything else that comes with a rare disease…
When Trey Kearns was two, he had a terrible itch. His itch wasn’t like yours - it was inside the body, meaning it's an itch you can’t scratch. They came…
Recently, biopharmaceutical company Vanda Pharmaceuticals announced that the FDA granted priority review status for a New Drug Application (NDA) and Supplemental New Drug Application (sNDA) for both liquid and…
According to a story from GlobeNewswire, the biopharmaceutical company CymaBay Therapeutics, Inc. recently announced encouraging topline findings from a phase 3 clinical trial. This study was testing seladelpar as a…
Just because we are staying home to avoid COVID-19 doesn't mean the medical field has stopped! In fact, many doctors are more determined than ever to improve patient outcomes.…
What do you call fossils that just lie there? Lazy bones! But for the Centrosaurus apertus, its bones are talking to us from beyond the grave, making it less lazy and…
As reported in Parkinson's News Today, a new study published in Family Medicine and Community Health has looked into the relationship between concussions and certain other neurological disorders that patients have…
A recent article in The American Journal of Managed Care (AJMC) heralds the approval of a third CAR T-cell therapy, called Tecartus. Kite Pharma’s cell-based gene therapy is the first…
Metformin is a popular drug used by type 2 diabetes patients. According to recent interviews conducted by reporters for Medical Xpress, a team of researchers conducted parallel rodent and…
A blood test that gives a more accurate diagnosis of Alzheimer’s disease was announced at the July 2020 International Conference of the Alzheimer’s Association. The Association estimates that approximately…
Because of COVID-19, many meetings and events have been moved online - but that does not mean the learning has stopped. During the virtual Psych Congress Elevate conference, which…
According to a story from GlobeNewswire, the biopharmaceutical company Ovid Therapeutics Inc. and Columbia University have recently entered a collaborative partnership that will be focused on developing gene-based treatments for…
Each year, around 14,000 U.S. deaths are attributed to ovarian cancer. Yet researchers have historically struggled to find an exact cause of the cancer, or to understand exactly how…
Happy Friday! Did you know that August is Stevens-Johnson Syndrome Awareness Month? It's a type of severe skin reaction that often involves admission to a burn unit for treatment. We've…
According to a story from Charcot-Marie-Tooth News, an all new protocol first described in the journal Brain Sciences could mark a significant advance in Charcot-Marie-Tooth disease research. The protocol allows for the…
According to a story from GlobeNewswire, the first patient has been dosed in a recent clinical trial testing the experimental therapy tasquinimod as a treatment for multiple myeloma, a rare…
A recent article published in EMS WORLD cautions that long QT syndrome (LQTS), a potentially lethal disease in young people, often remains undiscovered. An ECG can detect the disease,…
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