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.
In 1873, a Russian physician coined the term “multiple myeloma” after discovering a patient with eight different forms of bone marrow tumors. In the early years, patients were treated with…
Continue ReadingNew Treatment Options Have Doubled Survival in Multiple Myeloma
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is…
Continue ReadingCompassion Corner: A Doctor’s Compassion Saved Her Son’s Life
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…
Continue ReadingJohns 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.…
Continue ReadingDIY: 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)…
Continue ReadingIs Gene Editing The Path Forward to Treat Mitochondrial Diseases?
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…
Continue ReadingA 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…
Continue ReadingUCSF 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.…
Continue ReadingTransthyretin 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…
Continue ReadingFive Year Old Holly Was Diagnosed With Batten Disease
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…
Continue ReadingUniversity 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…
Continue ReadingCushing’s Syndrome: Causes, Symptoms, and Treatment
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…
Continue ReadingUCB 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…
Continue ReadingWorking 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…
Continue ReadingA 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…
Continue ReadingThe 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,…
Continue ReadingHave 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,…
Continue ReadingMyrtelle 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…
Continue ReadingThe Nanopore Sequencing Test Reduces Diagnosis From Decades to Days for Multiple Rare Disorders
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…
Continue ReadingBrain 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…
Continue ReadingPatients 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…
Continue ReadingHave 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…
Continue ReadingPacritinib 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…
Continue ReadingAstraZeneca Announces Phase III Results for HER2-Low Metastatic Breast Cancer
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…
Continue ReadingTwo 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…
Continue ReadingResults are in: Opdivo plus Cabometyx Offer Prolonged Overall Survival for Patients with Advanced Kidney Cancer
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