According to a story from pm360online.com, the gene therapy company Fibrocell Science, Inc., recently received an investment of $900,000 from the Epidermolysis Bullosa Medical Research Foundation (EBMRF) and the EB…
At Patient Worthy, we are dedicated to the covering the latest stories about rare diseases that appear in the news cycle. While this is an important part of our mission…
According to a story from curetoday.com, a recent Phase 2 trial has demonstrated that bortezomib (marketed as Velcade) has the potential to be an effective therapy for patients with mantle…
According to a story from Proactive Investors, the biotechnology company Conatus Pharmaceuticals recently reported that the company's drug Emricasan failed to achieve the primary endpoint in a Phase 2b clinical…
According to a story from the Greenock Telegraph, twelve year old Megan McGarva, who was born with CDLK5 deficiency, will soon have to face a serious spinal operation. She has…
According to a story from EurekAlert!, the 60th Annual Meeting of the American Society of Hematology featured a number of different presentations of the latest research from St. Jude Children's…
According to a story from PR Newswire, the Blueprint Medicines Corporation recently announced that latest results from a Phase 1 clinical trial of avapritinib as a treatment for advanced systemic…
According to a story from pm360online.com, the biopharmaceutical company Ultragenyx Pharmaceutical Inc., in collaboration with Kyowa Hakko Kirin Co. Ltd, and Kyowa Kirin International PLC, recently announced that injectable burosumab…
At Patient Worthy, we are dedicated to the covering the latest stories about rare diseases that appear in the news cycle. While this is an important part of our mission…
According to a story from Medical Xpress, the US Food and Drug Administration has recently issued a new warning for the multiple sclerosis drug alemtuzumab (marketed as Lemtrada). This warning…
According to a story from globenewswire.com, the biopharmaceutical company Marinus Pharmaceuticals, Inc., recently announced the presentation of Phase 2 clinical trial of the drug ganaxolone as a treatment for CDKL5…
According to a story from biotech-finances.com, the biopharmaceutical company miRagen Therapeutics, Inc., recently announced the release of data from a Phase 1 clinical trial of miR-155 inhibitor cobomarsen as a…
According to a story from ctvnews.ca, Paul Fitzpatrick of Ontario has been enduring severe eye pain following laser eye surgery for over twenty years before he committed suicide. The man's…
According to a story from PR Newswire, the 60th Annual Meeting of the American Society of Hematology has been generating a lot of press lately as many groundbreaking studies, research,…
According to a story from globenewswire.com, the biotechnology company Innate Pharma SA recently announced the latest data from a Phase 1 clinical trial of the company's investigational medical product IPH4102.…
Richard Holl of Transient Global Amnesia (TGA) Project 2018 has played a role in the development of a resource website for patients that have experienced transient global amnesia. The website…
According to a story from Market Screener, an analysis of real-world data (data gathered outside of the clinical setting) suggests that the drug eltrombopag, marketed as Revolade, offers substantial advantages…
According to a story from BioPortfolio, the global biopharmaceutical company Celgene Corporation recently announced the release of early data from the dose-escalation portion of a Phase 1/2 clinical trial of lisocabtagene…
According to a story from PR Newswire, presentations at the 60th annual American Society of Hematology Meeting and Exposition are highlighting the results of four large scale clinical trials that…
According to a story from BioSpace, the biotechnology company Tiziana Life Sciences Plc has recently announced that participant enrollment in its Phase 2a clinical trial has finished. This clinical trial…
According to a story from einnews.com, the cell therapeutics company American Gene Technologies (AGT) has recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation…
According to a story from Stanford News, a team of researchers affiliated with Stanford University have successfully developed a new technique for studying high-grade serous ovarian cancer, which is one…
According to a story from BioPortfolio, the drug developer Novartis recently announced that the US Food and Drug Administration (FDA) has granted the company's investigational gene therapy AVXS-101, also known…
According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company's latest…
According to a story from The San Diego Union-Tribune, the Pancreatic Cancer Prevention and Screening Clinic at UC San Diego Moores Cancer center has opened for the first time this…
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