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 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,…

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    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…

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    Personalized Medicine: A Drug Tailored for a Six Year Old With Batten Disease May Serve as a Template for Similar Rare Disorders

      Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…

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    Found: Pelizaeus-Merzbacher Disease, the Cause, and a Potential Cure

      According to a recent article published by the University of California San Francisco, Pelizaeus-Merzbacher disease (PMD) is a rare neurological disease affecting young boys. PMD fatalities may occur before the child…

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    Studies Reveal How Blood Flow and Oxygen Delivery Are Affected by Sickle Cell Disease (SCD)

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    Physician and investigator John Wood, MD, Ph.D. of Childrens Hospital of Los Angeles specializes in studying the challenges facing SCD patients. He has currently focused his attention on how oxygen delivery…

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    Clinical Trials Show Promising Results for Risdiplam in Treatment of Spinal Muscular Atrophy Types 1, 2, and 3

    According to a recent article in SMA News Today, risdiplam (formerly RG7916)  is in ongoing studies of patients with spinal muscular atrophy (SMA). The drug is an investigational medicine developed by Genentech…

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    The Phase 3 Trial in Diabetic Peripheral Neuropathy May Have Failed Due to a Critical Mistake

      FierceBiotech recently reported that Helixmith Co., based in Korea and previously known as ViroMed, coordinated a Phase 3 clinical trial involving five hundred diabetic peripheral neuropathy (DPN) patients. The…

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    Step Therapy Supposedly Minimizes Risk, but for Brittany it Caused Severe Complications

      The Providence Journal recently published an article by Brittany Ricci, a student at Brown University’s Alpert Medical School, detailing her year-long struggle to receive the treatment her doctor had prescribed…

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