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.
The COVID era emphasized the importance of maintaining a strong immune system. But what about people who are less fortunate? Johns Hopkins researchers recently announced some good news for…
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Johns Hopkins Researchers Use Primary Immunodeficiencies to Discover New Info About Immune System Cells
Roger Bliss, 68-year-old pulmonary hypertension (PH) patient, was diagnosed with chronic lymphocytic leukemia and small lymphocytic lymphoma. In addition, Roger has celiac disease. He recently interviewed with Pulmonary Hypertension News.…
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DIY: A Pulmonary Hypertension Patient Drains His Lungs at Home
Mitochondria are called the ‘batteries’ that send power to our body’s cells. They convert energy from food into forms that cells can utilize. Mitochondria contain 0.1% of mitochondrial DNA (mtDNA)…
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Is Gene Editing The Path Forward to Treat Mitochondrial Diseases?
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According to an article recently published in Business Wire, Global Genes, a leading advocacy group, released a progress report on rare diseases. The report includes data and studies as…
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A Report on the Current Status of Rare Diseases and Key Findings for 2021
Mutations in the KRAS gene are the most aggressive type of solid tumor. They are responsible for one out of every four cancer deaths. A recent article published by Genentech…
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UCSF Study Inhibits Mutations in the KRAS Gene as a Potential Cancer Therapy
In August of 2021, Intellia Therapeutics of Cambridge and its partner Regeneron Pharmaceuticals published a study reported in the Boston Globe, showing results of a single treatment using CRISPR-Cas9 technology.…
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Transthyretin Amyloidosis: CRISPR-Cas9 Technology Shows Results in a Single Treatment
Amanda Gilpin, a resident of Karratha in Western Australia, noticed that her little daughter occasionally would roll her eyes in response to some of her motherly advice. At times, she…
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Five Year Old Holly Was Diagnosed With Batten Disease
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According to a recent article in NeurosScience News, one percent of individuals over the age of sixty are at risk for Parkinson’s disease. Patients are generally diagnosed at a late…
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University Team Discovers Two New Early Parkinson’s Symptoms
During this year’s commemoration of Rare Diseases, Leonard Saig M.D. a director at Bnei Zion Haifa spoke about Cushing’s syndrome, also called hypercortisolism. Cushing's syndrome is rare and difficult to…
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Cushing’s Syndrome: Causes, Symptoms, and Treatment
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UCB Inc., a biopharmaceutical company, has announced positive results for its RAISE Phase 3 trial (NCT04115293) investigating zilucoplan against placebo. According to a recent article in Biospace, zilucoplan met…
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UCB Inc. Announces Positive Results for Phase 3 Myasthenia Gravis Trial
The following story was told to R29 Fertility Diaries by a 34-year-old mother and her husband. The newly married couple was anxious to be parents. Even after the loss of…
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Working Through the Maze of Genetic Odds to Protect Their Child From Spinal Muscular Atrophy (SMA)
In January of this year, a University of Connecticut (UConn) team administered the world’s first infusion of a new gene therapy as part of a long-awaited clinical trial. According to…
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A UConn Health Team Administers the World’s First Gene Therapy for Glycogen Storage Disease Type-1a
The war on opioids inadvertently became a war on severely ill patients. The "war" had to be declared. There were reactions from all fronts to the realization that deaths rose…
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The New CDC Guidelines Show Compassion for Patients In Severe Pain
Note: This is part two of a two part story. Don't forget to go back and read part one! Meghan O’Rouke, the author of the NYT best-selling book Invisible Kingdom,…
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Have You Ever Been Denied a Diagnosis And Told That Your Pain is Psychosomatic? Part Two
Myrtelle, via Business Wire News, reports the completion of Phase 1/2 investigating its gene therapy for Canavan disease (CD) which they have now expanded to treat younger patients. Thus far,…
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Myrtelle Announces the First-in-Human Trial of a Targeted Gene Therapy for Canavan Disease (CD)
Researchers from Australia’s Garvan Institute have joined with collaborators in Israel and the UK in identifying neuromuscular and neurological genetic diseases that have been difficult to diagnose due to the…
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The Nanopore Sequencing Test Reduces Diagnosis From Decades to Days for Multiple Rare Disorders
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WDBJ recently carried special news from Virginia Tech’s Fralin Biomedical Institute. Treatment has been identified for glioblastoma patients. Glioblastoma is a fatal disease resulting in death within fifteen months after…
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Brain Cancer: Revolutionary New Treatment for Glioblastoma Patients
Nasopharyngeal carcinoma consists of cancers originating in the throat and the nose. Due to the way the cancers are positioned, radiation therapy may not be an option. Also, a number…
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Patients with Nasopharyngeal Carcinoma May Benefit From the Chinese Herbal Medicine Shengmai Yin
Too often a woman’s heart disease is labeled as stress and anxiety. If it is an autoimmune disorder, it is labeled as depression. Ovarian cysts are overlooked. The Katz Institute…
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Have You Ever Been Denied a Diagnosis And Told Your Pain is Psychosomatic? (Part One)
Cancer Network recently featured a press release by CTI BioParma announcing that the FDA has given accelerated approval for pacritinib to treat patients diagnosed with myelofibrosis and severely low platelets…
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Pacritinib Receives Accelerated Approval for Myelofibrosis With Severely Low Platelet Count
AstraZeneca and Daiichi-Sankyo are participating in a global collaboration to develop Enhertu (trastuzumab deruxtecan) for the treatment of HER2-low metastatic breast cancer (cancer that has spread). Enhertu is an ADC…
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AstraZeneca Announces Phase III Results for HER2-Low Metastatic Breast Cancer
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Scientists at London’s University College and a team of scientists at Stanford University simultaneously made the same discovery about amyotrophic lateral sclerosis (ALS): the genetic variant in the protein UNC13A…
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Two ALS Studies May Unravel its Genetic Roots and Solve One of its Mysteries
Patients with advanced kidney cancer have a new option in a combination therapy that will afford them a longer and better life. Kidney cancer can be hard to cure, but…
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Results are in: Opdivo plus Cabometyx Offer Prolonged Overall Survival for Patients with Advanced Kidney Cancer
Nicole Burns’ great grandfather left her his house and a portion of his 100 acres. According to a recent article in STAT News, Nicole also inherited her great grandfather’s heart…
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A Family Has a Protective Gene That is Keeping Them Alive: Part Two
The question plaguing the geneticists about the Burns family was: how have they survived? In 2016, several geneticists theorized that certain people may have a good-gene bad-gene scenario taking place…
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A Family Has a Protective Gene That is Keeping Them Alive: Part One