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.
Biological engineers at MIT are using a new technique to study various organs and their interaction with our immune system. According to a recent article in Science Daily, the researchers…
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Organs-On- A-Chip: A Novel Approach to Studying Inflammatory Bowel Disease
Identifying biomarkers may be an improved method of classifying Alzheimer’s and Parkinson’s. As outlined in a recent article covered by Parkinson's News Today, both diseases are diagnosed for the most…
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Using Artificial Intelligence to Study Alzheimer’s and Parkinson’s Disease
859 words vs 970 words 8% An article recently published in Science Daily quotes the results of a study showing a favorable outcome for six out of nine x-linked chronic…
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A Favorable Outcome for CGD Patients Post Stem Cell Gene Therapy
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Kidney disease is following other disorders as a target for new and improved machine learning. As published in a recent edition of Science Daily, pathologists have often classified kidney diseases…
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Machine Learning Changes the Dynamics of Kidney Disease Diagnosis
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To celebrate Rare Disease Day and raise awareness for approximately seven thousand rare diseases, Dr. Francis Collins, director of the National Institutes of Health, recently interviewed a person he…
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He Faced Death Multiple Times From iMCD But David Fajgenbaum Will Not Give Up
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My American Nurse recently carried an uplifting article showing increasingly positive changes for cystic fibrosis (CF) patients. A network of over one hundred thirty centers throughout the U.S. provide…
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Cystic Fibrosis Patients Are Living Longer Thanks to Improved Clinical Practices and Therapies
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…
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Good News for Patients with Urothelial Carcinoma of the Urinary Tract (UTUC)
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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…
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A Discussion of Research Behind Neuromuscular Disorders
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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…
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A Chinese Study Finds Higher Risk of Relapse and Resistance to Therapy in AAV Patients With Lung Involvement
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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…
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Vaccines for Prostate Cancer: Still Struggling After All These Years
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.…
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A First for CRISPR: Editing a Gene in the Retinal Cells of a Leber Congenital Amaurosis Patient
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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…
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Antiphospholipid Syndrome (APS): Young Women are Five Times More Likely Than Men to Have This Rare Disease
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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…
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Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders
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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…
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ICYMI: She was Diagnosed with Hashimoto’s Thyroiditis but her Doctor Didn’t Know How to Treat It
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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…
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A Patient With Appendix Cancer was Refused Enrollment in Five Clinical Trials
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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…
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Mitochondrial Disease Patient’s Parents Lose Law Suit Against Four Doctors and Boston Children’s Hospital
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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.…
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A Baby Was Taken From His Parents Because of an Undiagnosed Osteogenesis Imperfecta
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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Seven Questions for a Rare Disease Warrior
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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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Neurodegenerative-Neuromuscular Diseases and Airway Clearance Therapy
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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.…
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This College Student Turned her Spastic Diplegic Cerebral Palsy into Projects for the Disabled
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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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ICYMI: PharmaCyte Biotech’s New Weapon Against Pancreatic Cancer: Cell-in-a-Box®
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…
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The EMA Grants PRIME Designation to Viralym-M To Treat Viral Infections Linked to Stem Cell Transplant
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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…
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Toxic Protein Identified: Closing in on Alzheimer’s and Neurodegenerative Diseases
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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How Living With Hashimoto’s & Lyme Disease Taught Me To Stand Up For Myself — And My Health
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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…
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Pancreatic Cancer: Researchers are Calling Patients’ Response to this Treatment “Unprecedented“