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.

    Transthyretin-Mediated Amyloidosis (ATTR-CM); After Nine Years Pfizer Wins FDA Breakthrough Designation

    It has been nine years since Pfizer acquired the drugs vyndagel and vyndamax. According to a report in Biospace, based on the FDA’s recent approval of the two drugs to treat transthyretin-mediated…

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    CRISPR Uncovers New Gene Fusion as Novel Drug Target for Brain, Ovarian and other Cancers

      CRISPR-Cas9, the genome editing tool, has caused quite a stir in the scientific community. According to a recent article in Science Daily, with results published in Nature Communications, CRISPR has…

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    An In-Depth Discussion About FDA Oversight of Diagnostic Tests and the Effect of Inaccurate Tests on Patients

      Newly diagnosed patients may decide to either receive medical treatment or forego treatment based on the results of their diagnostic test.  As recently reported in PEW, although it is…

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    Drug Start Up Founder Says Drugs Will Work For You– But They Don’t Know Why

    Although somewhat of an exaggeration, the title of this article falls in line with the thinking of Insitro’s founder, Daphne Koller. In a recent interview with Bloomberg, Koller said that…

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    A Look at Progress in Healthcare and a Glimpse into the Future

      The Asco Post recently interviewed Dr. Jean Pierre Bizzari, the Executive Vice President of Celgene, a multinational bioparmaceutical company. The interview covered various aspects of research and development (R&D)…

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    ALZHEIMER’S:  Scientists are Back to Square One; Will Bryostatin-1 be the Missing Link?

      Pharmaceutical companies have spent billions of dollars on Alzheimer’s research. The target has been the toxic protein, amyloid-beta. According to a recent article in NPR News, this theory was…

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    New Research With the Potential to Alleviate Symptoms of Parkinson’s, Alzheimer’s and Charcot-Marie-Tooth Disorders

      A recent article in the neuroscience section of the Medical Express reports the results of a study conducted by researchers in the Charcot-Marie-Tooth/Hereditary Neuropathy Center at internationally acclaimed Cedars-Sinai.…

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    MALARIA: Between One Million and Two Million Deaths Each Year Worldwide.  This Company is on Target to Treat Severe Malaria

    An article in Globe Newswire quotes National Institute of Health statistics for malaria as being between 300 to 500 million cases worldwide resulting in 1.5 to 2.7 million deaths annually. People living…

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    New Data on the Difference Between Plasma Cell Myeloma and Smoldering Myeloma Has Treatment Implications
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    New Data on the Difference Between Plasma Cell Myeloma and Smoldering Myeloma Has Treatment Implications

    A recent report that was published in the Swiss Medical Weekly outlines data gathered by a Swiss panel of experts on plasma cell myeloma. The report offers an update and…

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    Researchers Believe Lithium May be Key to Developing a Drug for Limb Girdle Muscular Dystrophy

    The News Hub at Washington State University recently carried an article describing initial success by researchers towards developing a drug for a rare form of muscular dystrophy. Several thousand people…

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    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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