Did you know that zebrafish, a tropical freshwater fish, are being more commonly used to model and study human diseases? Now, says Phys.org, researchers from the Garvan Institute of Medical…
In a recent press release, biopharmaceutical company Acceleron Pharma, Inc. ("Acceleron") announced that its investigational drug candidate sotatercept received Orphan designation from the European Commission (EC) for the treatment of…
According to a story from Markets Insider, the biotechnology company REGENXBIO Inc. has recently released data from its phase 1/2 clinical trial. This is interim data from cohorts 1 and…
In early December 2020, biopharmaceutical company Gilead Sciences, Inc. ("Gilead") shared updated results from a Phase 1b clinical trial evaluating magrolimab for patients with treatment-naïve acute myeloid leukemia (AML). Generally,…
If you've ever heard the term "levodopa" be discussed, it was probably in regards to Parkinson's disease. Currently, levodopa (L-DOPA) is the most commonly prescribed therapy for patients with Parkinson's.…
According to a story from Globe Newswire, the biopharmaceutical company Ultragenyx Pharmaceutical Inc. has recently announced that its investigational treatment UX701 has earned the Orphan Drug designation from the US…
In early December 2020, mRNA medicines company Arcturus Therapeutics Holdings Inc. ("Arcturus") announced that the company begun dosing participants within its Phase 1b clinical trial. Within the trial, researchers will…
Did you know The Orphan Disease Center (ODC, a center within the Perelman School of Medicine at the University of Pennsylvania) has a program to assist patient groups and families…
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