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.
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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On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…
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Scientists in four continents worked for over a decade on a massive project that has resulted in the most detailed picture of cancer ever produced. Researchers now have a huge…
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Gene therapy is the treatment of disease by way of the transfer of genetic material into cells. According to a recent article in Labiotech, an EU publication, gene therapy…
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According to a report in Myeloma Research News, the European Commission has recently granted authorization to market bortezomib, dexamethasone and thalidomide (collectively known as VTd) plus daratumumab to treat…
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The Halcyon machine holds an imaging mechanism that produces a high-quality CAT scan plus it houses a treatment unit that delivers radiation. According to a recent news release, approximately twenty-four…
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A recent press release by the global biopharmaceutical research company, AbbVie, reported a positive opinion by CHMP granted to VENCLYXTO® combined with obinutuzumab to treat chronic lymphocytic leukemia (CLL). A positive opinion by…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…
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According to an article in the Alzheimer’s publication Being Patient, participants who had been enrolled in the discontinued phase three trials of aducanumab will start receiving the drug as…
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According to a January 2020 release by Business Wire, the USFDA has awarded accelerated approval of Epizyme’s TAZVERIK™ in the treatment of advanced epithelioid sarcoma (ES). The approval was…
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Janssen R&D, a division of Johnson & Johnson (J&J), has partnered with the UK Biobank and others according to a recent news release by 3BL Media. It is noteworthy…
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Although ‘precision medicine’ has been mentioned in medical circles since 1999, according to a recent article in Pharmafile, technological advancements have now made it feasible. For years physicians have…
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