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 Genetic Engineering and Biology News, Washington University and University of Hawaii researchers have uncovered a possible drug development candidate in soil bacterium called…
Dr. Christopher Austin, a National Institute of Health Director for Transitional Sciences, spoke at last year’s virtual conference honoring Rare Disease Day. According to an article in the NIH Record, Dr.…
Korea began its use of effective therapies for idiopathic pulmonary arterial hypertension (IPAH) early in 2000. Idiopathic hypertension has no apparent cause. Yet there have been very few studies assessing…
Maria’s message is: “when in doubt, get a biopsy." Ten years ago, Maria Sylvia noticed a vertical streak on her thumbnail that at the time she thought was “cool." She…
Necroptosis, a type of regulated cell death, has been implicated in diseases such as inflammatory bowel disease, as well as neurodegenerative diseases. According to a recent article in News Medical…
Rise is a nonprofit organization that gives support to the disabled. Its services include rides by Lyft, the popular ride-sharing company. The funds are then reimbursed by their riders’ Medicaid…
According to information from the National Institute of Health, one in nine elderly people in the U.S. will be affected by Alzheimer’s disease (AD). A recent article in the Globe Newswire…
Every year almost half of the fourteen thousand patients who have received bone marrow or stem cell transplants will develop chronic graft-versus-host disease (cGVHD). Currently, there are no options…
Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood. It spreads rapidly. Therefore precise and swift treatment is critical. In a recent interview with Medical Life…
If you have not heard of craniosynostosis, it is understandable, as the disorder affects only one infant out of approximately 2500. A recent article published in the Washington News…
Professors Susan Treves and Francesco Zorzato, Basel University researchers, have recently presented the first potential approach to congenital muscle diseases. Their report was published in a recent article by Science…
In 1873, a Russian physician coined the term “multiple myeloma” after discovering a patient with eight different forms of bone marrow tumors. In the early years, patients were treated with…
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 COVID era emphasized the importance of maintaining a strong immune system. But what about people who are less fortunate? Johns Hopkins researchers recently announced some good news for…
Roger Bliss, 68-year-old pulmonary hypertension (PH) patient, was diagnosed with chronic lymphocytic leukemia and small lymphocytic lymphoma. In addition, Roger has celiac disease. He recently interviewed with Pulmonary Hypertension News.…
Mitochondria are called the ‘batteries’ that send power to our body’s cells. They convert energy from food into forms that cells can utilize. Mitochondria contain 0.1% of mitochondrial DNA (mtDNA)…
According to an article recently published in Business Wire, Global Genes, a leading advocacy group, released a progress report on rare diseases. The report includes data and studies as…
Mutations in the KRAS gene are the most aggressive type of solid tumor. They are responsible for one out of every four cancer deaths. A recent article published by Genentech…
In August of 2021, Intellia Therapeutics of Cambridge and its partner Regeneron Pharmaceuticals published a study reported in the Boston Globe, showing results of a single treatment using CRISPR-Cas9 technology.…
Amanda Gilpin, a resident of Karratha in Western Australia, noticed that her little daughter occasionally would roll her eyes in response to some of her motherly advice. At times, she…
According to a recent article in NeurosScience News, one percent of individuals over the age of sixty are at risk for Parkinson’s disease. Patients are generally diagnosed at a late…
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…
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