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 LLS Ground-Breaking Precision Medicine Approach Master Trial  BEAT AML Releases Third Year Test Results

      A recent posting by the Leukemia and Lymphoma Society references the Beat AML Master Trial. The trial evaluates targeted therapies to treat acute myeloid leukemia (AML) and was launched…

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    Rare Diseases: Only 10% of India’s 1.37 Billion People Have Health Insurance

    In about five years India’s population will exceed that of China which is currently ranked number one according to population. Contrast these numbers with the fact that only ten percent…

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    In-Patient Hospitalization vs. At-Home Programs: Will Doctors Be Making House Calls Again?

    Ash Clinical News recently interviewed Dr. Bruce Leff of Johns Hopkins in connection with “hospital-at-home”, one of the major changes occurring in hospital care. Dr. Leff and his team at…

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    New Drug Under Review by the FDA to Treat the Most Lethal Form of Spinal Muscular Atrophy

      According to an article in Biospace, the FDA is currently reviewing data from the Phase III STRIVE clinical trial for possible approval of Zolgensma as gene therapy for spinal…

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    New Personal Kinetigraph (PKG) Allows Parkinson’s Patients in the UK to Connect with their Specialists

      Although guidelines for Parkinson’s patients suggest that they visit a specialist every six months, according to a report in Parkinson’s News Today, the waiting period may be up to…

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    Artificial Intelligence(AI) and a Solution to Detect Familial Hypercholesterolemia

      Familial Hypercholesterolemia (FH) is often misdiagnosed as plain old high cholesterol because of overlapping symptoms such as elevated lipid levels. According to a recent article in MedicalView, FH is three…

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    New England Journal of Medicine (NEJM) Announces Results of A Unique Study of Hirschsprung Disease Affecting Infants

      An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…

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    The First Adeno-Associated Viral Gene Therapy for Huntington’s Disease Receives the FDA’s Fast Track Designation
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    The First Adeno-Associated Viral Gene Therapy for Huntington’s Disease Receives the FDA’s Fast Track Designation

      A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…

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    uniQure Receives FDA Fast Track Designation for AMT 130 Gene Therapy for the Treatment of Huntingtons Disease

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs,  announced that the U.S. Food and Drug…

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    New Drug Application on Track for LCFAOD A Rare Autosomal Recessive Disease

    A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…

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    Researchers in Sweden Studied 116,330 Twins and This is What They Discovered About Autoimmune Diseases

      According to a recent article from EurekAlert!, researchers used data from the Swedish Twin Registry and found that most of the 7 diseases they were studying were inherited.  The second finding…

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    An International Organization Dedicated to  Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals

    Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…

    Continue Reading An International Organization Dedicated to  Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals