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.

    A Family With the Most Significant History of Breast and Ovarian Cancer in the US
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    A Family With the Most Significant History of Breast and Ovarian Cancer in the US

      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…

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    New Approach Uses CRISPR-Cas9 to Boost Fetal Hemoglobin Production to Treat Blood Disorders
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    New Approach Uses CRISPR-Cas9 to Boost Fetal Hemoglobin Production to Treat Blood Disorders

      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…

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    Parents React to Novartis’ International “Baby Lottery” for Zolgensma with Hope But Also With Outrage

    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.…

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    Three Courageous Women With Multiple Sclerosis are Fighting the Disease by Running Marathons.
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    Three Courageous Women With Multiple Sclerosis are Fighting the Disease by Running Marathons.

    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…

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    Outcomes Differ in Studies Conducted for Hereditary Hemorrhagic Telangiectasia (HHT)

    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,…

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    VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy Received Positive Opinion by the (EMA) 

      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…

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    Donors are Needed Due To A Shortage of Platelets That Threatens A Young Child’s Life

    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…

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    Rare Epilepsy Updates:  One New Drug, Two Clinical Trials, and Orphan Drug Designation

      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…

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    A Blind Artist, A Motivational Speaker Who Can’t Talk, and a Single Mom with Rheumatoid Arthritis

      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…

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    Pediatric Cancer and the 70th Anniversary of the Leukemia & Lymphoma Society

      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…

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    After Five Insurance Coverage Denials, the Next Appeal Brought a Young Patient Closer to Receiving Treatment for Metachromatic Leukodystrophy

      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 Reading After Five Insurance Coverage Denials, the Next Appeal Brought a Young Patient Closer to Receiving Treatment for Metachromatic Leukodystrophy
    IYCMI: CALQUENCE Has Received US FDA Approval to Benefit Adults with CLL
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    IYCMI: CALQUENCE Has Received US FDA Approval to Benefit Adults with CLL

      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…

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    CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 
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    CRISPR/Cas9 Looks Promising for Transfusion Dependent Sickle Cell Disease and Beta Thalassemia Patients 

       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…

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    Researchers are Gaining a Better Understanding of Disease Development at the Cellular Level

      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.…

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