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.
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…
An article in PubMed dated October 8, 2021 outlines immune therapies treating myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) patients. Both cancers are hematologic malignancies that begin in the…
BeyondSpring Pharmaceuticals has issued a press release announcing that the FDA presented the company with a complete response letter pertaining to its drug plinabulin and granulocyte colony-stimulating factor (G-CSF). The…
This week the Guardian reported that a pig’s heart, which was genetically modified, had been transplanted into a patient by doctors who were trying to save his life. The patient was…
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…
Biogen Inc. recently announced data that included extended results from its earlier Phase 3b NOVA study on the administration of natalizumab and the evaluation of the drugs Tysabri® and Vumerity. …
Diagnosing amyloidosis (abnormal protein in tissue) is a complex process. Over eighty percent of amyloidosis patients are undiagnosed. The disease takes hold after the blood plasma cells in the bone…
Nonalcoholic steatohepatitis is a liver disease, causing fat to build up in the liver. The resulting inflammation may lead to liver cancer or cirrhosis (scarring) and possible liver failure. As…
Many people living in rural areas of the U.S. must drive a long distance to receive their medical treatments. According to Yahoo News, twenty-nine-year-old Leah Stavenhagen, now living and…
The recent FDA approval of Vyvgart (efgartigimod) for generalized myasthenia gravis (gMG) marks the first drug approval for Argenx. Vyvgart is designed specifically for adults who test positive for…
WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the…
Continued From Part One 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.…
ANCA-associated vasculitis (AAV) generally targets two enzymes, myeloperoxidase (MPO) or proteinase 3 (PR3) located in the granules of monocytes and neutrophils. These enzymes play a critical role in the…
Catherine Nettles Cutter, a professor at Penn State University, vividly recalls two years of her life spent trying to recover from a bodysurfing accident that resulted in the mysterious loss…
One 66-year-old patient who lives in Poughkeepsie, N.Y. received the same diagnosis from three doctors. Each doctor suggested that his symptoms pointed to temporomandibular (TMJ) joint pain. The patient and…
After two years of in vitro fertilization treatments, Amber Freed, Age 38, and her husband Mark Freed of Denver, Colorado welcomed their twin babies Riley and Maxwell into the world.…
Victoria Gray’s recent interview with NPR confirmed that her genetically modified blood cells have truly transformed her life. Victoria volunteered in the first-ever attempt in the U.S. to use CRISPR,…
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