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.

    Since 1982, Over 16 Million People Have Been Cured of Hansen’s Disease Worldwide, But a Major Struggle Remains in Brazil

      According to a report recently published in the Inter Press Service, since the advent of multidrug therapy in 1982, over sixteen million people have been cured of Hansen’s disease,…

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    CRISPR Genome Editing and Stem Cell Technology Uncovered the Cause of the Baby’s Heart Disorder

      Tatiana Legkiy was only two months old when she was rushed to a hospital in San Francisco after an echocardiogram showed that her heart was malfunctioning. A recent article in…

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    Doctors Are Unable to Diagnose Many Rare Diseases. Genome Sequencing is Changing That Scenario

    After twenty-two years physician Huang Chun-jung finally learned why his vision and hearing have fallen to thirty percent of normalcy, thanks to research conducted by the National Health Research Institutes…

    Continue Reading Doctors Are Unable to Diagnose Many Rare Diseases. Genome Sequencing is Changing That Scenario

    Huntsman Lab Research Team Discovers that Selinexor May Benefit Myelofibrosis Patients Who have No Other Curative Options

      Myelofibrosis (MF) comes under the heading of rare cancer. A recent article in Newswise, University of Utah, describes the disease as the failure of bone marrow to produce normal…

    Continue Reading Huntsman Lab Research Team Discovers that Selinexor May Benefit Myelofibrosis Patients Who have No Other Curative Options

    Are People With Chronic Kidney Disease Being Excluded From Clinical Trials and Access to New Therapies?

    Major European and international kidney associations have joined in an awareness campaign to stress the urgent need to include patients with chronic kidney disease in clinical trials. In a recent…

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    The Future Looks Brighter for Premature Babies Thanks to Stem Cell Research and Tissue Engineered Intestines

      Dr. Tracy Grikscheit is a leading surgeon in the field of tissue engineering. Dr. Grikscheit, together with her colleagues at Children’s Hospital Los Angeles, co-authored an article for the…

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    Accelerated Approval Granted to a Novel Drug Combination That Treats Patients with a Common Type of Non-Hodgkin Lymphoma

      Each year over eighteen thousand people in the United States are diagnosed with diffuse large B-cell lymphoma (DLBCL) a common type of non-Hodgkin lymphoma. A recent FDA news release…

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    Acute Lymphoblastic Leukemia: Miracles for Millions and the People Who Created Them

      According to a recent article in MedicalXpress, the story begins fifty years ago with the grim statistics that a child with acute lymphoblastic leukemia (ALL) would not survive longer than…

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    A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body
    source: pixabay.com

    A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body

      The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain. Check Orphan recently carried an article that had been…

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    The New 5F9 Antibody Looks Encouraging as it Targets MDS and AML Leukemia in a Phase 1b Clinical Trial

      The California Institute for Regenerative Medicine (CIRM) has funded a study by a company called Forty Seven, Inc. The clinical trial is investigating the benefits of a drug therapy for two cancers…

    Continue Reading The New 5F9 Antibody Looks Encouraging as it Targets MDS and AML Leukemia in a Phase 1b Clinical Trial

    Legal and Ethical Questions: What is Pharma’s Obligation to Pursue Development of a Promising but Non-Profitable Drug?

      Dr. Craig Klugman, a bioethics professor at DePaul University, expresses his opinion in this recent article in Columbia University’s Bioethics.com. Dr. Klugman begins with a discussion of Pfizer’s biological drug,…

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    The FDA’s Director of the Center for Drug Evaluation Discusses Accelerated Approvals

      In the mid-1980s the FDA was criticized for being slow in approving new drugs. In fact, a commission was formed as a result of the mounting criticism. Janet Woodcock,…

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    The FDA is Reversing Course on Unapproved Products Sold by Stem Cell Manufacturers

      The FDA’s “untitled letter” to R3 Stem Cell (R3), based in Scottsdale Arizona, cautioned R3 that the product they are marketing is considered a drug. It must, therefore, be…

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    The FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy

      The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the same disease. Jaci has a rare…

    Continue Reading The FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy