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.

    Postural Orthostatic Tachycardia Syndrome (POTS): An Underdiagnosed Condition That Doctors Said Was All in Her Head

    About one to three million people in the United States are living with postural orthostatic tachycardia syndrome (POTS). The disease affects one in one hundred teens and is more common…

    Continue Reading Postural Orthostatic Tachycardia Syndrome (POTS): An Underdiagnosed Condition That Doctors Said Was All in Her Head

    Using PacBio SMRT Sequencing in Myotonic Dystrophy Type 1 and Other Rare Disorders

      Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…

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    Phase 3 CONDOR Study of PYL: The Potential To Improve The Outcome of Recurrent Prostate Cancer

    490 words 8% vs 1306 words 3% According to a recent Globe Newswire release, Progenics Pharmaceuticals, an oncology company that specializes in artificial intelligence and targeted therapies, announced results for its Phase…

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    CMS is so Rare That it Took Four Years to Enroll Twenty People for a Trial

      According to a recent article in Biospace, Catalyst Pharmaceutical’s CMS-001 Phase 3 study ended with mixed results. The trial was billed as the first placebo-controlled double-blind study testing amifampridine phosphate (brand…

    Continue Reading CMS is so Rare That it Took Four Years to Enroll Twenty People for a Trial

    The First Clinical Evidence that the Drug Mavoglurant Improves Responsiveness in Fragile X Syndrome (FXS)

    Results of a study reported in PLOS ONE that was conducted by a team of scientists indicate that the experimental drug mavoglurant improves responsiveness and eye gaze for patients with…

    Continue Reading The First Clinical Evidence that the Drug Mavoglurant Improves Responsiveness in Fragile X Syndrome (FXS)

    First Patient to Receive Gene Therapy in a Phase 1/2 Study of ACTUS-101 in Patients with Pompe Disease

    Actus Therapeutics, a privately held portfolio company of AskBio, has recently announced through PRWeb, the initiation of patient dosing in a clinical trial testing its investigational gene therapy ACTUS-101. About Pompe…

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    After 120 Drug Failures, Can this be the Drug to Slow Alzheimer’s Progression?

      Over the years Alzheimer’s patients and their families have seen more than one hundred twenty drugs that were developed for the treatment of Alzheimer's have failed. For a while,…

    Continue Reading After 120 Drug Failures, Can this be the Drug to Slow Alzheimer’s Progression?
    New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects
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    New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects

      CNN recently covered a story in the publication Nature about a paper explaining base editing (or prime editing). The researchers who created the technology set forth the process of using base…

    Continue Reading New Prime Editing Has The Potential to Search and Replace up to 89% of Genetic Defects

    The Open Targets Initiative Brings Scientists Closer to Identifying the Cause of Autoimmune Diseases

      Scientists believe that “it is in our DNA.” According to a recent article in EurekAlert, a five-year study formulated through Open Targets together with the Sanger Institute and their…

    Continue Reading The Open Targets Initiative Brings Scientists Closer to Identifying the Cause of Autoimmune Diseases