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.
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Over the years Alzheimer’s patients and their families have seen more than one hundred twenty drugs that were developed for the treatment of Alzheimer's have failed. For a while,…
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A guest speaker at the 2019 Rare Disease Forum recently held at the North Carolina Biotechnology Center acknowledged that screening is necessary to justify the need for a particular…
CNN recently covered a story in the publication Nature about a paper explaining base editing (or prime editing). The researchers who created the technology set forth the process of using base…
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According to a recent article in Horizon, the World Health Organization estimates that every one couple out of ten is dealing with fertility issues. The long-held assumption has been…
A recent article in Medical Life Science News reports that a recent study has discovered a new mechanism that interacts with an immune cell receptor called Mincle. This receptor regulates…
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A recent article in the Science Daily reports on a study that demonstrated in detail how the human immune system located in the brain is altered during certain diseases such…
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Scientists believe that “it is in our DNA.” According to a recent article in EurekAlert, a five-year study formulated through Open Targets together with the Sanger Institute and their…
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Lately, there have been many newsworthy articles about Novartis and Zolgensma (onasemnogene abeparvovec-xioi). In May 2019 the FDA approved Zolgensma, a one-time treatment for the most severe form of…
Jessica Walton described the staff at Queen Elizabeth Hospital in King’s Lynn as being in “a bit of a panic” when they thought that she was having a stroke.…
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A recent article in Forbes highlights a study on diagnostic performance published in The Lancet Digital Health. The study represents the first review to compare the diagnostic accuracy of deep…
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Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…
Supplies of a Critical Bladder Cancer Drug are Dwindling Bob Field, a 72-year-old New York banking executive, received a call from his urologist’s office canceling his next appointment. According to…
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According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…
Physician and investigator John Wood, MD, Ph.D. of Childrens Hospital of Los Angeles specializes in studying the challenges facing SCD patients. He has currently focused his attention on how oxygen delivery…
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According to a recent article in SMA News Today, risdiplam (formerly RG7916) is in ongoing studies of patients with spinal muscular atrophy (SMA). The drug is an investigational medicine developed by Genentech…
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Waukesha Wisconsin Police Captain Dan Baumann recently held a news conference that was described in an article published in Scientific American. Captain Baumann said that parents of a teenage boy showed…
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FierceBiotech recently reported that Helixmith Co., based in Korea and previously known as ViroMed, coordinated a Phase 3 clinical trial involving five hundred diabetic peripheral neuropathy (DPN) patients. The…
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Pamela Smith, age 67, had run out of options when her melanoma metastasized and became inoperable. According to a recent article in MedicalXpress, in January 2014, Pamela joined the…
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The Providence Journal recently published an article by Brittany Ricci, a student at Brown University’s Alpert Medical School, detailing her year-long struggle to receive the treatment her doctor had prescribed…
A new treatment was recently tested for blastic plasmacytoid dendritic cell neoplasm (BPDCN). A recent article in MD Anderson News points out that in the U.S., five hundred to…
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Richard Engel’s son Henry, age 4, has been diagnosed with Rett syndrome, a thus far incurable genetic neurological disease. Engel is chief foreign war correspondent at NBC and has…
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A molecular biologist, Dr. Daria Julkowska, coordinates an international consortium in Paris that is largely funded through the 28-member European Union (EU). According to a report published in the Charcot-Marie-Tooth…
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Alzheimer’s disease (AD) is a neurodegenerative disease and the major cause of impaired memory affecting the older population. Reported cases are rapidly increasing. So far, clinical trials have failed…
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The accelerated approval granted by the FDA for the Duchenne muscular dystrophy drug eteplirsen, developed by Sarepta Therapeutics, was granted only after internal disagreements and protests had been resolved.…
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Fierce Biotech recently focused on a report by The BMJ, a British science journal, on the validity of clinical trials which are critical to decision making by patients and…
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