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.
Lithium boosts muscle strength in mice with rare muscular dystrophy New drug target identified Click to share on Pinterest (Opens in new window) Removing one gene caused normal muscle muscle…
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The mesothelioma diagnosis was a surprise to Raya Bodnarchuk of Baltimore, Maryland because she had never worked in a factory, mine, the military, or a shipyard. These are the typical…
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A birth involving mitochondrial replacement therapy took place as part of a clinical trial in Greece according to a recent article in STAT. Greece is now the second country…
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Familial Hypercholesterolemia (FH) is often misdiagnosed as plain old high cholesterol because of overlapping symptoms such as elevated lipid levels. According to a recent article in MedicalView, FH is three…
A three-year research grant of $11 million has been awarded through the Gilbert Family Foundation in America. According to a recent article in News-Medical Life Sciences, the Foundation was formed…
According to a recent article in Check Orphan, genetic and biochemical testing are combined in a new method of metabolic testing called the CentoMetabolic panel. The announcement comes from…
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An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…
A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…
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An article in Austin Gastroenterology recently asks this question. It also confirmed what we already know, that genetic testing is now available to the average person and in fact,…
LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug…
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An encouraging article recently published in Sickle Cell Anemia News announced that due to the urging of pharmaceutical companies and patient advocacy groups, the FDA has produced an updated draft…
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The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the FDA based on interim results of the clinical trial ADMIRAL, according to an article in EurekAlert for Science News. A…
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OncLive recently published the views of several speakers at its State of the Science Summit on breast cancer. Stephanie LaBomascus, MD gave a presentation that highlighted triple negative breast…
The oral inhibitor drug gilteritinib (XOSPATA®) was recently approved by the USFDA based on interim results of the clinical trial ADMIRAL according to an article in EurekAlert for Science News. A total of 371 adult patients…
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A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…
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A Rare Intervention For His Children’s Rare Disease An inspiring article has recently been published in the journal Nature that credits Nick Sireau, a resident of the UK, with…
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According to a recent article from EurekAlert!, researchers used data from the Swedish Twin Registry and found that most of the 7 diseases they were studying were inherited. The second finding…
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Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…
A group of international scientists has been exploring using new DNA-based nanomachines for gene therapy. The nanomachines are hoped to be more effective in the treatment of oncological diseases. As…
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A recent article in the journal Nature reports that Phase 1 GAPVAC-101 First-In-Human trial is one of the first clinical trials with a goal of designing and manufacturing actively personalized vaccines…
M ost of the time, Whitney Weldon doesn’t think about her second skeleton. She was 9 the first time it really flared up. Bridges of bone grew from nowhere to…
418 words 9% vs 744 words 5%Researchers in Sweden Studied 116,330 Twins and This is What They Discovered About Autoimmune DiseasesAccording to a recent article in the EurekAlert researchers used data from…
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A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…
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A recent article in Parkinson’s News Today reports that a reduction in a patient’s required medication and management of motor system disorders may be achievable with Deep-Brain Stimulation (DBS) for…
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Following a record number of approvals in 2018, the FDA has recently approved two new drugs with many more anticipated approvals later in the year. Trazimera to Treat Human Epidermal…
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