On September 30, 2020, BioCryst Pharmaceuticals ("BioCryst") announced promising data on their drug candidate BCX9930. The therapy, an orally administered Factor D inhibitor, is designed to treat patients with previously…
Recently, biopharmaceutical company Apellis Pharmaceuticals announced submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for pegcetacoplan. If approved, these applications will allow for patients with…
As reported in BioSpace; today, Biocryst Pharmaceuticals has been given a boost to further their work to explore the merits of a potential oral treatment for PNH. The U.S. FDA…
On August 31, 2020, biopharmaceutical company Omeros Corporation announced that it will soon move forward with clinical trials for OMS906. This decision follows clearance of an Investigational New Drug Application…
According to a story from globenewswire.com, the biopharmaceutical company Apellis Pharmaceuticals, Inc. has announced that it is currently making plans to submit a New Drug Application (NDA) to the US…
The UFC has some tough fighters in it. But 35-year-old Joe Ellenberger never thought that his most difficult opponent would be within himself. That is, his paroxysmal nocturnal hemoglobinuria (PNH),…
According to a story from Barron's, the biotechnology company Alexion Pharmaceuticals has recently announced plans to test its drug Ultomiris as a potential treatment for COVID-19. As the scientific community…
According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company's investigational drug…
Regeneron Pharmaceuticals has recently announced the results of Phase 2 of their study of pozelimab, which is an experimental treatment for paroxysmal nocturnal hemoglobinuria. Current therapies for this condition…
According to a story from Market Screener, the biopharmaceutical company Alexion Pharmaceuticals has recently announced some encouraging news regarding the status for ravulizumab (marketed as ULTOMIRIS) in the EU. The…
Paroxysmal nocturnal hemoglobinuria (PNH) prematurely destroys the bodies red blood cells (hemolysis). It can cause severe anemia, kidney disease, fatigue, as well as other symptoms. Up until now, there has…
Big news out of the FDA last week! Alexion Pharmaceuticals got approval for ALXN1210 for paroxysmal nocturnal hemoglobinuria, or PNH, which will now be sold as Ultomiris, while Stemline got approval…
According to a story from Morningstar, the company Alexion Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has agreed to review their Biologics Licensing Application for their…
According to a story from Business Wire, the company Alexion Pharmaceuticals recently announced that it has submitted a Biologics License Application (BLA) to the FDA for the approval of one…
On December 11, Alexion Pharmaceuticals, Inc. released dose-ranging data from a recent clinical study involving treatment with ALXN1210, which is an investigational long-acting C5 complement inhibitor meant to treat sufferers…
The U.S. Food and Drug Administration (FDA) is quite an impressive agency. They regulate the safety and security of all foods that are produced, imported, or created in any other…
Wouldn’t it be nice if you could talk to one of those “nationally known” specialists about your aplastic anemia? Well, if you can make it to West Palm Beach, Florida…
Paroxysmal nocturnal hemoglobinuria, or PNH, is a hematopoietic stem cell disorder. Hematopoietic stem cells are developed in the bone marrow, where they grow and eventually develop into red blood cells, white…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
