When Jessica Kalnas was growing up, her mother Judy thought her daughter was just clumsy. The youngest of six kids, Jessica was always falling over: off of her bike,…
As published by the Food and Drug Administration; The U.S. FDA has just approved the first FDA approved therapy derived from cannabis (Epidiolex, a CBD solution) to treat tuberous sclerosis…
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…
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