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.
Medscape Medical News recently published an article heralding the POUT clinical trial (NCT01993979). The trial brought evidence that patients who received platinum-based chemotherapy after surgery saw their risk of…
A recent editorial focusing on NMDs (neuromuscular disorders) was published in the journal Disease Models & Mechanisms. The editorial highlights a special issue of the journal devoted to building…
A recent article in ANCA Vasculitis News highlighted a Chinese study of ANCA-associated vasculitis (AAV), a group of autoimmune diseases. An earlier Chinese study suggested that the risk of…
The term ‘failure’ may no longer apply to cancer vaccines according to a recent article in Marketwatch. Researchers have endured over four decades of frustration since the first two…
NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…
According to a recent article in Healio, the National Institute of Health states that one-third of strokes occurring for the first time to people under fifty can be linked to…
According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
Susan Mann was seven years old when she was diagnosed as having Hashimoto’s thyroiditis, an autoimmune disease that affects the thyroid. According to a recent article in the online…
Todd Mercer, a fifty-two year old Michigan resident and father of two, recently called into STAT’S Podcast The Readout LOUD to discuss the difficulty he has had getting enrolled…
The Boston Globe recently interviewed the principals in a case brought by the Pelletier family against the Boston Children’s Hospital. The article gives details about a family’s charge of…
The charges against Baby Jace’s parents, Crystal Bryant (age 24) and Jarvis Bryant (age 27), seemed to imply that they were guilty until two doctors took a closer look.…
Caption: David Fajgenbaum (right) and I pose for a photo a few years ago in Philadelphia. Credit: National Disease Research Interchange, Philadelphia Tomorrow is Rare Disease Day at NIH, marking…
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Advances in medicine have been responsible for longer overall survival in patients with neurodegenerative-neuromuscular diseases. According to a recent report in RTMagazine, an increase in the number of patients…
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Jennifer Schlegel’s life began earlier than expected with an unanticipated disease. According to an article in The Lantern, Jennifer was born prematurely and diagnosed with spastic diplegic cerebral palsy.…
Cell-in-a-Box, the name of one of Biotech’s new products is just as intriguing as the drug itself. A recent article in NewsFile characterizes the product as having the potential…
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According to a recent article in The Pharma Letter, the European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) designation to Viralym-M based on positive results from a Phase 2 proof-of-concept…
For years scientists have tried to understand how and why tau, well known to researchers for its implication in Alzheimer’s disease, changes from its normal form to a harmful…
February 16, 2020 The doctor wasn't listening to me. He didn't care that I was suffering. “I’m so sorry, I thought we were doing the right thing,” my dad said,…
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The ASCO Post recently reported findings of high response rates and improved survival from the combination of platinum-based cisplatin and gemcitabine (chemotherapy) in BRCA/PALB2 mutation positive pancreatic ductal adenocarcinoma. The goal…
The publication Being Patient tracks the latest news about Alzheimer’s disease. This month it published an article about a new Alzheimer’s clinical trial that is being conducted with the…
One of the wealthy regulars on the TV show Shark Tank made his millions by starting a home-based business selling tee shirts. An article in Bridgeport’s ctPost publication chronicles…
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ALS News Today recently announced the introduction of a Phase 3 clinical trial for oral edaravone, a drug that has previously been approved for slowing the decline of physical…
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According to a recent article in ALS News Today, the FDA’s Orphan Status designation has been granted to NeuroSense Therapeutics for its drug, PrimeC, to treat ALS. PrimeC received the designation…
Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…
An article in CURE® describes two extremely rare types of tumors: paraganglioma and pheochromocytoma. One in three thousand individuals may have either or both of these tumors. The symptoms accompanying…
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