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.

    Vamorolone Has Been FDA Approved to Treat Duchenne Muscular Dystrophy
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    Vamorolone Has Been FDA Approved to Treat Duchenne Muscular Dystrophy

    Vamorolone, now marketed as AGAMREE®, is termed a dissociative steroid therapy, as it has proven its potential to retain the muscle-strengthening and anti-inflammatory benefits of corticosteroids. On the other side…

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    Mom Signed Up Thousands of Stem Cell Donors in Support of Son with IPEX
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    Mom Signed Up Thousands of Stem Cell Donors in Support of Son with IPEX

      Every three to four minutes, someone in the U.S. is diagnosed with a blood disease such as lymphoma, leukemia, or sickle cell anemia. As reported in the Pittsburgh Post-Gazette,…

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    Study Shows an Average of 4 1/2 Minutes of Vigorous Exercise May Reduce the Risk of Cancer by 32%

    The words ‘vigorous exercise’ usually bring to mind running marathons or playing pickleball. Inside Precision Medicine reports that researchers at a Sydney, Australia University tracked the daily activity of more…

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    Wealthy Facioscapulohumeral Muscular Dystrophy Patient Invests $100 Million to Find a Cure  

      Chip Wilson, billionaire founder of Lululemon Athletica, was first diagnosed with muscular dystrophy in 1987 at the age of 32. Wilson, who is now 66 years old told Global…

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    Encouraging Findings in Phase 2 Gastric Cancer Clinical Trial
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    Encouraging Findings in Phase 2 Gastric Cancer Clinical Trial

      Arcus Biosciences and Gilead Sciences issued a press release announcing encouraging overall response rates and six-month progression-free survival rate for their combination treatment of domvanalimab plus zimberelimab and chemotherapy…

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    Scientists Demonstrate Gut Microbiome Connection with Transfer of Alzheimer’s to Healthy Animals
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    Scientists Demonstrate Gut Microbiome Connection with Transfer of Alzheimer’s to Healthy Animals

      Scientists have previously suggested that the gut microbiome plays a role in causing Alzheimer’s disease, and this has now been confirmed. A recent report from ScienceAlert covered a study…

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    Promising Interim Data in Phase 1/2 Hunter Syndrome Trial
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    Promising Interim Data in Phase 1/2 Hunter Syndrome Trial

    Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…

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    Wave Life Science’s New Trial Application is Causing Renewed Excitement Around RNA Editing
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    Wave Life Science’s New Trial Application is Causing Renewed Excitement Around RNA Editing

      If Wave Life Sciences’ recently submitted trial application for WVE-006 is approved, it will become the first RNA editing treatment in a clinical development setting. Wave’s CEO, Paul Bolno,…

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    Infantile Spasms: Mom Saves Baby’s Life After Being Dismissed by Two Hospitals
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    Infantile Spasms: Mom Saves Baby’s Life After Being Dismissed by Two Hospitals

    Imagine the frustration of Faustina Cavero, unable to follow her maternal instincts that told her something was wrong with her baby. Doctors at two different hospitals disagreed with her and…

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    Scientists Are Calling Monkey’s Two-Year Survival After a Pig Kidney Transplant “Unprecedented”
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    Scientists Are Calling Monkey’s Two-Year Survival After a Pig Kidney Transplant “Unprecedented”

      The xenotransplant involved transplanting Yucatan miniature pig kidneys into macaque monkeys with 69 genetic modifications. One monkey survived for over two years, offering hope to the team of Harvard…

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    Researchers Seek Reason for a Higher Survival Rate in Obese Patients with Pulmonary Arterial Hypertension (PAH)

    Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…

    Continue Reading Researchers Seek Reason for a Higher Survival Rate in Obese Patients with Pulmonary Arterial Hypertension (PAH)
    Two CTCL Trails on Partial Hold After Patient Death
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    Two CTCL Trails on Partial Hold After Patient Death

      The two trials evaluating lacutamab, an investigational therapy developed to treat T-cell lymphomas, were put on partial hold by the FDA. The patient died of hemophagocytic lymphohistiocytosis, which is…

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    Over 200 Rare Diseases to be Included in Genomics England Research Study
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    Over 200 Rare Diseases to be Included in Genomics England Research Study

    Genomics is revolutionizing healthcare processes, offering the potential to enhance the lives of numerous individuals by enabling the early detection of treatable disorders and providing lifesaving therapies. Every year, thousands…

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    Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy
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    Death of Muscular Dystrophy Patient Potentially Caused by Adeno-Associated Virus That Delivered Gene Therapy

    For over four decades, the aspiration of gene therapy has been to develop novel therapies with the potential to enhance human health. Recombinant AAV (rAAV), a type of gene therapy,…

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