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 Long Road to FDA Approval of Plinabulin and G-CSF to Prevent Chemotherapy Induced Neutropenia

    BeyondSpring Pharmaceuticals has issued a press release announcing that the FDA presented the company with a complete response letter pertaining to its drug plinabulin and granulocyte colony-stimulating factor (G-CSF). The…

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    Korean Group’s Successful Experiment to Treat Nonalcoholic Steatohepatitis
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    Korean Group’s Successful Experiment to Treat Nonalcoholic Steatohepatitis

    Nonalcoholic steatohepatitis is a liver disease, causing fat to build up in the liver. The resulting inflammation may lead to liver cancer or cirrhosis (scarring) and possible liver failure. As…

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    Compassion Corner: Compassionate Use, Expanded Access Part Two: The Terminology Is Different But the Unmet Need Is the Same
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    Compassion Corner: Compassionate Use, Expanded Access Part Two: The Terminology Is Different But the Unmet Need Is the Same

    Continued From Part One 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.…

    Continue Reading Compassion Corner: Compassionate Use, Expanded Access Part Two: The Terminology Is Different But the Unmet Need Is the Same
     Two Recent FDA and EMA Approvals Bring Hope to ANCA Associated-Vasculitis Patients
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     Two Recent FDA and EMA Approvals Bring Hope to ANCA Associated-Vasculitis Patients

       ANCA-associated vasculitis (AAV) generally targets two enzymes, myeloperoxidase (MPO) or proteinase 3 (PR3) located in the granules of monocytes and neutrophils. These enzymes play a critical role in the…

    Continue Reading  Two Recent FDA and EMA Approvals Bring Hope to ANCA Associated-Vasculitis Patients
    Compassion Corner: Compassionate Use, Expanded Access Part One: The Terminology Is Different But the Unmet Need Is the Same
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    Compassion Corner: Compassionate Use, Expanded Access Part One: The Terminology Is Different But the Unmet Need Is the Same

    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 a…

    Continue Reading Compassion Corner: Compassionate Use, Expanded Access Part One: The Terminology Is Different But the Unmet Need Is the Same

    A New Drug to Treat Focal Segmental Glomerulosclerosis and Renewed Hope for Vertex Pharmaceuticals

    After several clinical setbacks, Vertex Pharmaceuticals found promising results in its study of a drug to treat a genetic kidney disease called focal segmental glomerulosclerosis (FSGS). As reported recently by…

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     A Clinical Trial Confirms That Nivolumab Plus Chemotherapy Outperforms Immunotherapy Alone In Gastric Cancer Treatment

    Healio recently published results of the CheckMate 649 study confirming the benefits of combining nivolumab and chemotherapy in treating newly diagnosed patients with gastroesophageal junction cancer and advanced gastric and…

    Continue Reading  A Clinical Trial Confirms That Nivolumab Plus Chemotherapy Outperforms Immunotherapy Alone In Gastric Cancer Treatment
    Chronic Granulomatous Disease: A Stem Cell Transplant Approach to Speed up the Wait List
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    Chronic Granulomatous Disease: A Stem Cell Transplant Approach to Speed up the Wait List

      According to Dr. Deepak Chellapandian, a Johns Hopkins specialist in bone marrow transplant for rare diseases, the prognosis for his patient was very poor. Jocelyn was born with chronic…

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    CRISPR/Cas9 Gene Editing Adds Ultrasound Therapy for Hepatocellular Carcinoma to its List of Possibilities
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    CRISPR/Cas9 Gene Editing Adds Ultrasound Therapy for Hepatocellular Carcinoma to its List of Possibilities

       Washington University Researchers readily acknowledge the attributes of sonodynamic therapy (SDT) as a promising therapeutic method to treat hepatocellular carcinoma (HCC).  SDT is noninvasive with high tissue penetration, but…

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    Dr. Richard Stone Discusses Development of Eight Drugs for Acute Myeloid Leukemia Within Four Years
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    Dr. Richard Stone Discusses Development of Eight Drugs for Acute Myeloid Leukemia Within Four Years

      Dr. Richard Stone recently interviewed with Targeted Oncology to discuss challenging acute myeloid leukemia subtypes and a list of available therapies. Dr. Stone was asked to look back at…

    Continue Reading Dr. Richard Stone Discusses Development of Eight Drugs for Acute Myeloid Leukemia Within Four Years