United Therapeutics Corporation has recently presented data from the Phase 3 INCREASE trial, which evaluated Tyvaso as a treatment for pulmonary hypertension associated with interstitial lung disease. Results were presented…
According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…
According to a story from UT Health San Antonio, a team of researchers have discovered in a new study the mechanism behind the activation of magnesium ions in cells. The…
As many patients with rare diseases know, it can sometimes be difficult to achieve the correct diagnosis. In fact, the average length of time it takes from symptom onset to…
A recent study published in the Journal of Translational Medicine describes an accurate and objective approach that aims to evaluate the severity of myalgic encephalomyelitis, also known as chronic fatigue syndrome. This…
Patricia Simon Patricia Simon was born with a cleft lip and palate. She is now a nurse, a public speaker, a patient advocate, and a writer. She has a great…
According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…
According to a recent article in ScienceAlert, scientists have conducted experiments in mice activating a protein called protrudin that gives damaged nerve fibers the ability to regenerate. Currently, once nerve…
In a recent press release, biopharmaceutical company Rallybio announced that the first participant was dosed in a Phase 1/2 clinical trial. Currently, only healthy male participants are enrolled in the…
After announcing positive results from a Phase 2b ADVISE trial, Arena Pharmaceuticals, Inc. ("Arena") hopes to soon initiate a Phase 3 clinical trial to evaluate etrasimod as a treatment…
Recently, biopharmaceutical company Everest Medicines ("Everest") announced positive topline data from the Phase 3 NefIgArd trial, which was run by its licensing partner Calliditas Therapeutics AB ("Calliditas"). Together, the…
According to a new study, researchers believe that lower limb functional surgery improves walking ability and quality of life (QOL) in patients with Charcot-Marie-Tooth disease (CMT). This option provides a…
The European Medicines Agency has accepted Biogen's Marketing Authorization Application (MAA) for review. The application is for aducanumab, a treatment for Alzheimer's disease. As this condition faces an unmet medical…
While clinical trials can be extremely helpful in the development of new treatments, not all trials go as planned. Recently, biotechnology company Regeneron Pharmaceuticals ("Regeneron") discovered this firsthand. As the…
On November 3, 2020, biopharmaceutical company ChemoCentryx and global pharmaceutical company Vifor Pharma Group's sub-business Vifor Fresenius Medical Care Renal Pharma (VFMCRP) announced that the European Medicines Agency will review…
In early November, global biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") announced the acceptance of its New Drug Application (NDA) for vosoritide. The investigational injectable treatment is designed to treat pediatric…
Later this week, the American Association for the Study of Liver Diseases (AASLD) will hold its virtual The Liver Meeting Digital Experience (TLMdX). Altogether, TLMdX will last from November 13-16.…
In late October, molecular information company Foundation Medicine, Inc. ("Foundation") shared that its diagnostic tool FoundationOne CDx was approved as a companion diagnostic for VITRAKVI (larotrectinib). While FoundationOne CDx will…
Pancreatic cancer is a rare but severe cancer, and it is often difficult to diagnose until it has advanced. When you combine this with a lack of knowledge and awareness…
Tuberous sclerosis complex (TSC) is a rare disease that, like many other rare diseases, is not yet fully understood by medical professionals. Because of this, it is very difficult to…
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…
Bryan and Bradford Manning recently gave an interview to the Associated Press. The brothers told the story of their vision loss as a result of a rare degenerative eye disease.…
According to a story from PR Newswire, a new collaboration between the Hairy Cell Leukemia Foundation (HCLF) and the Leukemia & Lymphoma Society (LLS) will aim to support critical research…
A recent article in Science News highlighted a discovery by Richard Seifes, M.D. of Baylor College of Medicine. Dr. Seifes has discovered the origin of a liver disease that…
For nearly all of 2020, news of COVID-19 dominated the headlines. The first U.S. lockdowns associated with the viral pandemic occurred in March. Now, in November, the novel coronavirus…
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