Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.
In a recent article appearing in BioPortfolio, Orphan Technologies announced a Phase 1/2 clinical trial involving the first Classical Homocystinuria (HCU) patients to be treated with OT-58. Twelve patients were enrolled as…
A recent article in CheckOrphan reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the synthetic GLP-2 analog apraglutide for the treatment of short bowel syndrome…
ANCA Vasculitis News recently reported findings published in the journal Arthritis and Reumatology on the study “Efficacy and Safety of Belimumab and Azathioprine for Maintenance of Remission in ANCA‐Associated Vasculitis: A…
In a story published in The Hernando Sun, Dawn Dybiec, age 31, describes her disbelief when she and her husband were told that her two sons, Gabriel and Steven, were…
The Florey Institute of Neuroscience (FIN), one of the world’s largest and most respected brain research centers, recently announced trial results for the drug CuATSM, developed by FIN, the School…
David Radford, a 63-year-old resident of Oxford, UK, and a liver cancer survivor, said his surgeon attributes his successful liver transplant to the new normothermic perfusion machine. As reported recently…
Pharnext SA, a biopharmaceutical company, recently announced results in a PLOS ONE article entitled "Early short-term PXT3003 combinational therapy delays disease onset in a transgenic rat model of Charcot-Marie-Tooth disease 1A…
UPI Health News recently reported that a study, published in the New England Journal of Medicine, showed that fecal microbiota transplantation resulted in a 32 percent remission rate in patients…
Patients with acquired thrombotic thrombocytopenic purpura (aTTP) showed a significantly improved platelet count response after treatment with Cablivi® according to the New England Journal of Medicine's Phase 3 trial results. A…
A January 4th post by the Lancet's EBioMedicine headlined the first-in-human pilot study results published by UT Health San Antonio in collaboration with the Mayo Clinic and the Wake Forest School…
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Stephanie Hosford was recently interviewed by CURE (Cancer Updates Research and Education) about what she now calls her “three crazy days”. The title of her book is “Bald, Fat and…
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