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.
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…
A year ago, Paddy Doherty’s doctor told him that he had a rare hereditary disease called transthyretin (ATTR) amyloidosis, the same disease that had killed his father. As reported in…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Research by three doctors provided a basis for tyrosine kinase inhibitors (TKI) and offered advancements in treatment for renal cell carcinoma (RCC) patients. Two such TKIs, sunitinib and sorafenib, remain…
Positive results were announced today by Anavex Life Sciences, which is based in New York, for its Phase 3 clinical trial of Anavex 2-73. Participants in the trial were female…
Researchers are aware that Black cancer patients experience worse outcomes than white cancer patients due to the differences in access to health care. According to a report in Cancer Network,…
Recent clinical trials have proven that treatment for relapsed ovarian cancer is not a standard “one size fits all." After a relapse, areas where tumors spread and grow in the…
Targeted Oncology recently featured a discussion by Dr. Jennifer Brown, director of Dana Farber’s Chronic Lymphocytic Leukemia Center, on the interim results from the SEQUOIA Phase 3 trial. The trial…
It is common practice to treat multiple myeloma patients with a stem cell transplant. In the GENESIS trial, the stem cells were autologous (from the patient’s own peripheral blood or bone…
For the last seven years, the Global Genes Rare Compassion Program has been providing medical students with an opportunity to bond with patients and families who have been diagnosed with…
U.S. News carried an article reporting that in a recent study, researchers have identified minute amounts of cancer (organoids) in fallopian tube tissue. The significance of the discovery is that…
Professor Miranda Orr of Winston Salem’s School of Medicine in North Carolina leads a team of investigators who have detected senescent cells in patients with Alzheimer’s disease. These cells, found…
The World Health Organization (the WHO) announced World Leprosy Day which this year will be celebrated on January 30th. The theme of the celebration is “United for Dignity.” It…
Julia Lefelar, a resident of Gaithersburg, MD, was telling a friend about her years of seeing different doctors trying to find the cause of her illness. After a while, her…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
Early detection is the key to treating so many types of cancer. Autoimmune hepatitis (AIH), chronic inflammation of the liver, is brought about by an immune system disorder. AIH must…
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