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.
According to a recent article in the Sunderland Vibe, the MRC Developmental Pathway Funding Scheme (MRC) was created to carry out the work of Professor Roz Anderson, who recently died of…
In the early 1900s, German psychiatrist Alois Alzheimer, while studying about the decline in brain function, recognized a correlation between behavioral changes and the buildup of tangles and plaques in…
Most biological drugs, including insulin, can't be taken orally, because enzymes and proteins cause the drugs to break down too quickly in the digestive system. For decades, the pharma industry…
Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…
Massimo Federico, MD, director of the medical oncology unit at the University of Modena and Reggio Emilia, Italy, was quoted in Cancer Network as saying that “Achieving a complete clinical and…
The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…
A recent article in CheckOrphan describes a study that was conducted by Researchers at the Lysosomal and Rare Disorders Research and Treatment Center in Vancouver, B.C. The study examines the…
Alexion Pharmaceuticals, Inc., a company that is advancing a rare disease pipeline in complement biology, recently issued a series of press releases announcing positive results of a Phase 3 study…
Ofatumumab as a Single Agent Results of ofatumumab administered as a single agent with an extended induction (initial treatment) brought about a high response rate and it was well tolerated among follicular…
The treatment of metastatic renal cell carcinoma (RCC) has evolved over the last ten years and investigators now look towards combination therapies to improve response rates and durability for…
The Food and Drug Administration (FDA) recently announced its approval of Venetoclax (Venclexta) in combination with either decitabine or azacitidine (hypomethylating agents) or cytarabine (an antimetabolite) to treat adults 75 years…
“Doctor” Onelio Hipolit-Gonzalez has been charged with unlicensed practice of a health care profession and unlawful use of a two-way communication device according to an article published in the Spectrum News of…
A report published in 2018 in The Hemotologist mentions the fact that most of the therapies introduced over the years for acute myeloid leukemia (AML) simply meant optimizing the use of…
The University of Louisville’s publication UL News recently reported a finding by its research team regarding an immune checkpoint molecule. The molecule had been developed for cancer immunotherapy but when…
A recent interview with David Alvarez and his mother in the CTPost explains how he manages to remain positive after being diagnosed with spinal muscular atrophy (SMA). David is a freshman…
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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