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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Myelofibrosis (MF) comes under the heading of rare cancer. A recent article in Newswise, University of Utah, describes the disease as the failure of bone marrow to produce normal…
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Major European and international kidney associations have joined in an awareness campaign to stress the urgent need to include patients with chronic kidney disease in clinical trials. In a recent…
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The Center for Disease Control (CDC) is urging doctors and researchers to investigate and to collect new data before the disease strikes again. An article in Ars Technica reports that…
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Gov.UK recently published a speech by Baroness Blackwood in which she announced that the National Health Service (NHS) is currently involved in one of the most significant transformations in social…
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Duchenne muscular dystrophy (DMD) is the most common, yet severe, form of muscular dystrophy, a group of rare neuromuscular disorders. DMD is caused by mutations in the gene that codes…
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The FDA is putting patients with rare diseases front and center knowing that these patients have little support and in many cases cannot find specialists to diagnose and treat their…
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Dr. Tracy Grikscheit is a leading surgeon in the field of tissue engineering. Dr. Grikscheit, together with her colleagues at Children’s Hospital Los Angeles, co-authored an article for the…
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The first indication of the influence that people can have in clinical trials when acting as a community may have occurred in the 1980s. As reported recently in Viral…
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Kris Newby and her husband were unaware that they both had been bitten by ticks while on vacation on a small ”Vineyard” island. An article that appeared recently in…
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Each year over eighteen thousand people in the United States are diagnosed with diffuse large B-cell lymphoma (DLBCL) a common type of non-Hodgkin lymphoma. A recent FDA news release…
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According to a recent article in MedicalXpress, the story begins fifty years ago with the grim statistics that a child with acute lymphoblastic leukemia (ALL) would not survive longer than…
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Social media, and especially Facebook, have become our “windows to the world”. According to a recent article that appeared in The Michigan Public Health News Center, scientists are using…
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The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain. Check Orphan recently carried an article that had been…
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The California Institute for Regenerative Medicine (CIRM) has funded a study by a company called Forty Seven, Inc. The clinical trial is investigating the benefits of a drug therapy for two cancers…
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Fifty years ago, the air base in Ho Chi Minh City, formerly Saigon, was the world’s busiest airport. The base is now surrounded by four neighborhoods with about 111,000…
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Dr. Craig Klugman, a bioethics professor at DePaul University, expresses his opinion in this recent article in Columbia University’s Bioethics.com. Dr. Klugman begins with a discussion of Pfizer’s biological drug,…
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In the mid-1980s the FDA was criticized for being slow in approving new drugs. In fact, a commission was formed as a result of the mounting criticism. Janet Woodcock,…
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The FDA’s “untitled letter” to R3 Stem Cell (R3), based in Scottsdale Arizona, cautioned R3 that the product they are marketing is considered a drug. It must, therefore, be…
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Health News Digest recently published an article that describes a simple blood test called DELFI, meaning DNA evaluation of fragments for early interception. Its simplicity belies the fact that it can detect…
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The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the same disease. Jaci has a rare…
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Ron Davis and his gene-sequencing technologies were mentioned years ago in The Atlantic alongside Elon Musk (SpaceX) and Jeff Bezos of Amazon fame. Since then, Davis has amassed over…
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Calls to Ban CRISPR A scientist named He Jiankui recently shocked the scientific world with his announcement that he had created twin “CRISPR babies”. The babies, a result of…
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It has been nine years since Pfizer acquired the drugs vyndagel and vyndamax. According to a report in Biospace, based on the FDA’s recent approval of the two drugs to treat transthyretin-mediated…
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CRISPR-Cas9, the genome editing tool, has caused quite a stir in the scientific community. According to a recent article in Science Daily, with results published in Nature Communications, CRISPR has…
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Newly diagnosed patients may decide to either receive medical treatment or forego treatment based on the results of their diagnostic test. As recently reported in PEW, although it is…
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