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.
Ty Eschenbaum’s world of high school football and working on his family’s ranch and farm changed suddenly after his freshman year when he was diagnosed with leukemia. Ty, who…
Continue ReadingLeukemia Survivor Arranges Make-a-Wish Trip for a Young Caroli Disease Patient
According to an article in BioSpace, the FDA recently approved the PARP inhibitor Lynparza that was developed by Merck and AstraZeneca to be used as maintenance therapy for pancreatic…
Continue Reading2020 Brings Hope to Patients with Pancreatic Cancer Thanks to Newly Approved Lynparza
Susan Winn’s family history of ovarian and breast cancer can be traced back to the 1860s. Susan’s adult daughters, Kathryn, Bridget and Maureen, were recently interviewed with CURE® (Cancer Updates Research…
Continue ReadingA Family With the Most Significant History of Breast and Ovarian Cancer in the US
An article published earlier this year by Fierce Biotech outlines an approach that was developed by researchers at the Fred Hutchinson Cancer Research Center. Using CRISPR-Cas9’s gene-editing method the researchers were…
Continue ReadingNew Approach Uses CRISPR-Cas9 to Boost Fetal Hemoglobin Production to Treat Blood Disorders
According to a recent article in Euronews, this year one hundred children who were diagnosed with spinal muscular atrophy (SMA) will receive Zolgensma, the world’s highest-priced drug, at no cost.…
Continue ReadingParents React to Novartis’ International “Baby Lottery” for Zolgensma with Hope But Also With Outrage
According to recent articles in Runner’s World Magazine, having a diagnosis of multiple sclerosis has not stopped Cheryl Hile, Jennifer Lee or Melissa Ossanna. Each woman is fighting the symptoms…
Continue ReadingThree Courageous Women With Multiple Sclerosis are Fighting the Disease by Running Marathons.
According to the UK’s National Institute for Health Research (NIHR), a new technology called machine perfusion (MP) has the potential to solve organ shortage, a major challenge in liver transplantation.…
Continue ReadingLiver Transplants Are Vastly Improved By A New Technology
An open letter to The Editor from Gaetani et al was recently published in the Orphanet Journal of Rare Diseases. Dr. Gaetani and associates at the Fondazione Policlinico Universitario, Rome Italy,…
Continue ReadingOutcomes Differ in Studies Conducted for Hereditary Hemorrhagic Telangiectasia (HHT)
Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the European Medicines Agency’s (EMA) Committee for…
Continue ReadingVYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy Received Positive Opinion by the (EMA)
Tahnee Hamilton was recently interviewed by KSL-TV about her young child, Avery, who was diagnosed less than a year ago with T Cell Acute Lymphoblastic Leukemia. About Veno-Occlusive disease (VOD) Avery…
Continue ReadingDonors are Needed Due To A Shortage of Platelets That Threatens A Young Child’s Life
CNN reported details of Ed’s illness as related by his fiancée Katy Berteau, who remained at this side continually despite risking her own health. Katy and Ed had planned to…
Continue ReadingESPN Reporter Edward Aschoff Died On Christmas Eve, His 34th Birthday, of a Rare Disease
According to a recently published article in Targeted Oncology, the results of the Phase III CANDOR trial were presented at the 61st ASH annual conference in Orlando, Florida. The…
Continue ReadingThis Three Drug Combination for Multiple Myeloma Looks Promising
A recent article published in Globe Newswire heralded Marinus Pharmaceuticals’ new drug, ganaxolone. The drug has been studied in over 1600 patients and is now being tested in two clinical…
Continue ReadingRare Epilepsy Updates: One New Drug, Two Clinical Trials, and Orphan Drug Designation
At times there seems to be no happy medium in life. A recent article in Bloomberg claims that the speed at which the FDA is approving newly developed drugs is…
Since most people cannot truly understand the challenges faced by a blind person, then an artist who cannot see is unimaginable. According to a recent article in the British…
Continue ReadingA Blind Artist, A Motivational Speaker Who Can’t Talk, and a Single Mom with Rheumatoid Arthritis
When the Leukemia & Lymphoma Society (LLS) was founded in 1949 by a family in memory of their sixteen-year-old son, cancer was considered a hopeless condition. As noted recently…
Continue ReadingPediatric Cancer and the 70th Anniversary of the Leukemia & Lymphoma Society
According to a recent article in the ASH Clinical News patients are being encouraged to participate in shared decision-making (SDM) with their doctors, nurses and other medical personnel. The…
Continue ReadingA New Patient-Centered World is Emerging in Hematology
Encouraging results from a clinical study were reported recently in Science & Enterprise describing a treatment for ALS using stem cells extracted from a patient’s bone marrow. The clinical…
Continue ReadingA New Experimental Treatment Brings Improvement to ALS Patients
A recent article in Bloomberg’s SFGate focused on the price of drugs. The average patient cannot afford the price of many drugs now on the market. Insurance companies have…
Continue ReadingFour Doctors Who Worked on the Development of Tafamidis Criticize its Price
On Dec 3, 2019 Share A blind artist, a motivational speaker who can’t speak and a young mom with arthritis: These are just three of the British Columbians shattering stereotypes…
Continue ReadingMeet 3 British Columbians shattering stereotypes about their disabilities – My City News
A recent article in the Taunton Gazette describes the dilemma faced by the parents of four-year-old Jaxtien Miller who is a metachromatic leukodystrophy patient waiting for a stem cell…
Continue ReadingAfter Five Insurance Coverage Denials, the Next Appeal Brought a Young Patient Closer to Receiving Treatment for Metachromatic Leukodystrophy
A recent article in BioPortfolio carried AstraZeneca’s announcement of the FDA’s approval of CALQUENCE® (acalabrutinib) for adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The approval…
Continue ReadingIYCMI: CALQUENCE Has Received US FDA Approval to Benefit Adults with CLL
CRISPR/Cas9 technology reached another milestone with encouraging results in two recent trials of CTX001. An article in Pharmaceutical Technology carried an announcement by CRISPR Therapeutics and Vertex Pharmaceuticals about…
Continue ReadingCRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients
Status Update: Shared Decision-Making in Hematology Sunday, December 1, 2019 Beth Faiman, CNP, PhD Certified nurse practitioner at the Cleveland Clinic in Cleveland, Ohio In this edition, ASH Clinical News…
Continue ReadingStatus Update: Shared Decision-Making in Hematology
The University of Southern California (USC) recently published an article in USC News announcing that researchers have discovered a technique allowing them to untangle the coiled, spiraling DNA molecules.…
Continue ReadingResearchers are Gaining a Better Understanding of Disease Development at the Cellular Level
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