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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Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…
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Researchers at McGill University celebrated a “first” by creating a mouse model in a dish to demonstrate the effect that mutations in the SLC9A6 gene have on brain cells.…
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The number of people in the U.S. who are permanently disabled or who die each year as a result of misdiagnosis is estimated to be over 100,000. EurekAlert recently reported…
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Many patients may not be aware of the vital role they play in medical discoveries, or that without their participation there would be no medical discoveries. CheckOrphan recently presented…
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Genomic studies and advances in technology have increased researchers’ knowledge of several genetic syndromes that put people at risk for acute myeloid leukemia (AML). An article that appeared in…
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According to a report recently published in the Inter Press Service, since the advent of multidrug therapy in 1982, over sixteen million people have been cured of Hansen’s disease,…
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Tatiana Legkiy was only two months old when she was rushed to a hospital in San Francisco after an echocardiogram showed that her heart was malfunctioning. A recent article in…
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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…
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After twenty-two years physician Huang Chun-jung finally learned why his vision and hearing have fallen to thirty percent of normalcy, thanks to research conducted by the National Health Research Institutes…
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We often see reports about the latest superbug in the news. Superbugs affect approximately two million people each year and of those, about 23,000 people will not survive. The…
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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…
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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Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
