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.

    Researchers are Gaining a Better Understanding of Disease Development at the Cellular Level

      The University of Southern California (USC) recently published an article in USC News announcing that researchers have discovered a technique allowing them to untangle the coiled, spiraling DNA molecules.…

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    Her Baby’s SLC6A1 Genetic Disorder is Currently Untreatable, But Amber Freed is Committed to Finding a Cure

      Amber’s determination to find a miracle for her son Maxwell is detailed in a recent BuzzFeed article. Maxwell and his twin sister, Riley, were born in March 2017. For…

    Continue Reading Her Baby’s SLC6A1 Genetic Disorder is Currently Untreatable, But Amber Freed is Committed to Finding a Cure

    Dr. Marc Bonaca Presents Insight Into Future Trends for Cardiovascular Disease and the Need For Teamwork

    Dr. Marc Bonaca, a cardiologist with the Brigham and Women's Hospital in Boston, recently gave a presentation at the Las Vegas 2019 VIVA conference on trends in vascular disease. As reported in…

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    A Father is Asking for Worldwide Birthday Cards for His Seven-Year-Old Daughter Lyla Who Is Scheduled for her 10th Brain Operation

      An article in the Hartlepool Mail announced that Lyla O’Donovan, her parents, and her four brothers and sisters will be celebrating Lyla’s seventh birthday on November 28th, 2019 at…

    Continue Reading A Father is Asking for Worldwide Birthday Cards for His Seven-Year-Old Daughter Lyla Who Is Scheduled for her 10th Brain Operation
    ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK
    source: pixabay.com

    ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK

      An article appeared recently in BioNews announcing the first-ever approval of an RNA-based therapy for use on the British National Health Service. The name of the drug is Patisiran…

    Continue Reading ICYMI: hATTR Amyloidosis was Untreatable Until a Gene Silencing Drug was Approved in the UK

    Hartlepool dad’s worldwide birthday card appeal for daughter Lyla O’Donovan, who is facing tenth brain operation

    The family of a little girl who is battling health problems after suffering a brain tumour are appealing for people to make her birthday extra special by sending her cards…

    Continue Reading Hartlepool dad’s worldwide birthday card appeal for daughter Lyla O’Donovan, who is facing tenth brain operation

    “Do not be an island”: Teamwork prescribed to tackle major demographic trends in vascular disease

    Teamwork prescribed to tackle major demographic trends in vascular disease 13th November 2019 207 Marc Bonaca addresses delegates at VIVA 2019 “Caring for vascular patients is going to take a…

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    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…

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    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…

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    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)