Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

    The FDA Will Allow the Most Expensive Therapy in the World to Remain on the Market

    The headline in a recent Washington Post article declared that the FDA claimed Novartis, through its newly-acquired company AveXis, manipulated data in an application for a gene therapy drug. These…

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    Researchers are Working to Repair a Small Step In a Pathway Involved in Neurodegenerative Diseases

    It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

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    This Time a “First-of-its-Kind” Study is “Out of this World” on Board the Space Station

      As reported recently in PhysOrg by NASA, researchers aboard SpaceX CRS-18 cargo flight are focusing on what triggers Parkinson’s disease and multiple sclerosis, both neurodegenerative diseases. Researchers suspect that…

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    This Young Man Accurately Diagnosed His Own Disorder But Doctors Did Not Believe His Diagnosis

    Doug Lindsay ran track in high school. According to an article published by CNN Health, Doug had big plans as a senior at Rockhurst University in Kansas City where he…

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    A Brief Discussion of How Cell and Gene Therapies are Being Improved for Future Generations

      While the last few years have seen major advances in all areas of the pharmaceutical industry, some of the newest and brightest talents are now focusing on cell and…

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    DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

    Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…

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    Research Team Identifies Diagnostic Errors in Three Classes of Disease Associated with a High Death Rate or Permanent Disability

      The number of people in the U.S. who are permanently disabled or who die each year as a result of misdiagnosis is estimated to be over 100,000. EurekAlert recently reported…

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    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…

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    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…

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    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

    514 words 10% vs 1741 words 3% Dr. Tracy Grikscheit is a leading surgeon in the field of tissue engineering. Dr. Grikscheit, together with her colleagues at Children’s Hospital Los…

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