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.
During this year’s commemoration of Rare Diseases, Leonard Saig M.D. a director at Bnei Zion Haifa spoke about Cushing’s syndrome, also called hypercortisolism. Cushing's syndrome is rare and difficult to…
UCB Inc., a biopharmaceutical company, has announced positive results for its RAISE Phase 3 trial (NCT04115293) investigating zilucoplan against placebo. According to a recent article in Biospace, zilucoplan met…
The following story was told to R29 Fertility Diaries by a 34-year-old mother and her husband. The newly married couple was anxious to be parents. Even after the loss of…
In January of this year, a University of Connecticut (UConn) team administered the world’s first infusion of a new gene therapy as part of a long-awaited clinical trial. According to…
The war on opioids inadvertently became a war on severely ill patients. The "war" had to be declared. There were reactions from all fronts to the realization that deaths rose…
Note: This is part two of a two part story. Don't forget to go back and read part one! Meghan O’Rouke, the author of the NYT best-selling book Invisible Kingdom,…
Myrtelle, via Business Wire News, reports the completion of Phase 1/2 investigating its gene therapy for Canavan disease (CD) which they have now expanded to treat younger patients. Thus far,…
Researchers from Australia’s Garvan Institute have joined with collaborators in Israel and the UK in identifying neuromuscular and neurological genetic diseases that have been difficult to diagnose due to the…
WDBJ recently carried special news from Virginia Tech’s Fralin Biomedical Institute. Treatment has been identified for glioblastoma patients. Glioblastoma is a fatal disease resulting in death within fifteen months after…
Nasopharyngeal carcinoma consists of cancers originating in the throat and the nose. Due to the way the cancers are positioned, radiation therapy may not be an option. Also, a number…
Too often a woman’s heart disease is labeled as stress and anxiety. If it is an autoimmune disorder, it is labeled as depression. Ovarian cysts are overlooked. The Katz Institute…
Cancer Network recently featured a press release by CTI BioParma announcing that the FDA has given accelerated approval for pacritinib to treat patients diagnosed with myelofibrosis and severely low platelets…
AstraZeneca and Daiichi-Sankyo are participating in a global collaboration to develop Enhertu (trastuzumab deruxtecan) for the treatment of HER2-low metastatic breast cancer (cancer that has spread). Enhertu is an ADC…
Scientists at London’s University College and a team of scientists at Stanford University simultaneously made the same discovery about amyotrophic lateral sclerosis (ALS): the genetic variant in the protein UNC13A…
Patients with advanced kidney cancer have a new option in a combination therapy that will afford them a longer and better life. Kidney cancer can be hard to cure, but…
Nicole Burns’ great grandfather left her his house and a portion of his 100 acres. According to a recent article in STAT News, Nicole also inherited her great grandfather’s heart…
The question plaguing the geneticists about the Burns family was: how have they survived? In 2016, several geneticists theorized that certain people may have a good-gene bad-gene scenario taking place…
Dr. Rachel Grisham MSK ovarian cancer section head discussed clinical trials investigating PARP inhibitors for ovarian cancer patients. The event was a round table discussion and interview for Targeted Oncology.…
Tay-Sachs disease has escaped effective treatment since 1883 when an ophthalmologist names Warren Tay found small red spots in the back of the eyes of some infants. According to an…
Stoke Therapeutics and Acadia Pharmaceuticals made a joint announcement this week through Business Wire of their collaboration in the development of RNA-based medicines to potentially treat rare neurodevelopmental disorders of…
CURE® recently conducted an interview with von Hippel-Lindau (VHL) patient Joseph Heisler and his mother, Marirene. Joseph’s father was diagnosed thirty years ago with the genetic (inherited) disease. Shortly thereafter…
Dr. Douglas Marchuk, professor of genetics at Duke, has spent years studying the genetics of cardiovascular disease. According to a recent article in the Atlantic, his research at the University…
Continued From Part One Case #2 The second case involved a sixteen-year-old male in the ICU unit with suspected bacterial hypoxic respiratory failure associated with viral pneumonia. The teen’s history…
The International Consortium of FD/MAS invites you to its Film Screening and Community Discussion on the 24th of February at 7:30 pm EST. The event is free and is in…
A group of autoimmune diseases called anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) is the subject of a preprint under consideration at Pediatric Rheumatology. ANCA-AAV is characterized by damage to small…
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