According to Healio, there is now another health-related issue that can cause more hospitalization and worse quality of life (QOL) in patients with pulmonary arterial hypertension (PAH): obesity. In the…
Since its conception, biopharmaceutical company Geron Corporation ("Geron") has worked to develop first-in-class therapies for hematologic myeloid malignancies. More recently, Geron began evaluating imetelstat for patients with lower risk myelodysplastic syndromes…
Pumpkin pie, ginger-bread cookies, candy canes, and chocolates. Oh what a sweet season! A few nights ago my kids and I spent a few hours crouched over the wafting smells…
Dan Hopkins is a bowler who has recently raised over $21,000 for cystic fibrosis (CF) in a 65 hour long bowl-a-thon. While he may be exhausted and sore, he is…
Admittedly, 2020 has been tough for a variety of reasons. One of the greatest? The spread of COVID-19, a novel coronavirus attributed to 72.8 million cases and 1.62 million deaths…
Currently, there are a host of health-related issues associated with diabetes. One specific health problem, which currently represents an unmet need, is pain associated with diabetic peripheral neuropathy. According to…
If you are like me and have complete inability to regulate temperature, then you might be looking for some awesome accessories to make life a little less painful this winter…
According to a recent press release, oncology company Novocure enrolled its first patient in a Phase 3 clinical trial to evaluate Optune, alongside radiation therapy, for patients with newly diagnosed…
Author: Harsha K Rajasimha, MS, Ph.D. Editor: Sharlene Brown, Ph.D. When clinical investigators and study coordinators start planning a new study design, speed and efficiency are paramount. Running a clinical…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Thinking about the year-end holidays brings up a kaleidoscope of images, and an array of emotions for me. And everyone else too, I imagine. There are the vivid memories of…
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…
For most of 2020, the coronavirus pandemic has dominated the headlines. What began earlier this year has transformed into a viral crisis with 69.8 million cases worldwide. To stifle the…
Crystal Goodwin, from South Portland, Maine, was diagnosed with mast cell activation syndrome (MCAS) back in 2011. A rare disease diagnosis can turn one's life upside down, and Crystal is…
Xencor, a biopharmaceutical company that focuses on curating treatments for cancers and autoimmune conditions, has recently released data on vibecotamab. This acute myeloid leukemia treatment was included in a Phase…
According to a story from Cancer Therapy Advisor, a recent phase 1 clinical trial investigated an experimental CAR T-cell therapy in heavily pretreated patients living with multiple myeloma, a rare…
As of last month, a newborn screening advisory committee from the Virginia Department of Health declined to add Krabbe disease to the conditions that doctors look for when a child…
In a recent press release, biotechnology company HemoShear Therapeutics ("HemoShear") shared that the company, in collaboration with Takeda Pharmaceutical Company Ltd. ("Takeda") identified a novel therapeutic target for nonalcoholic steatohepatitis…
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