Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.
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Popular Science published an article recently about the first time an attempt was made to treat sickle cell anemia using CRISPR technology. The patient, Victoria Gray, age 41, from…
The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…
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It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…
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As reported recently in PhysOrg by NASA, researchers aboard SpaceX CRS-18 cargo flight are focusing on what triggers Parkinson’s disease and multiple sclerosis, both neurodegenerative diseases. Researchers suspect that…
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Doug Lindsay ran track in high school. According to an article published by CNN Health, Doug had big plans as a senior at Rockhurst University in Kansas City where he…
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While the last few years have seen major advances in all areas of the pharmaceutical industry, some of the newest and brightest talents are now focusing on cell and…
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As set forth in a recent MD Anderson Cancer Center publication, targeted therapy makes it possible to personalize cancer treatment. It is also known as “precision medicine”. For about forty…
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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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514 words 10% vs 1741 words 3% Dr. Tracy Grikscheit is a leading surgeon in the field of tissue engineering. Dr. Grikscheit, together with her colleagues at Children’s Hospital Los…
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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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