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 FDA has Approved the First Oral Drug for Maintenance Therapy of High-Risk Neuroblastoma
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    The FDA has Approved the First Oral Drug for Maintenance Therapy of High-Risk Neuroblastoma

    Neuroblastoma most commonly occurs in children under the age of five. The disease usually affects areas of the adrenal gland (positioned over the kidneys) or areas in the neck, chest,…

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    Mother Hosts Moonlight Ball to Help Her Daughter with Xeroderma Pigmentosum
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    Mother Hosts Moonlight Ball to Help Her Daughter with Xeroderma Pigmentosum

      Health professionals continually warn the public to take steps to guard against sunburn. We often hear the expression “one in a million.” The phrase and the warning truly pertain…

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    Alzheimer’s: Another Drug Failure Has the Researchers Questioning the Science
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    Alzheimer’s: Another Drug Failure Has the Researchers Questioning the Science

      Roche has added another failure this year with its drug gantenerumab. As noted in Fierce Biotech, the drug was unable to improve functional or cognitive decline in two Phase…

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    Experts Are Comparing the Threat of Bacterial Antimicrobial Resistance (AMR) to the HIV/AIDS Health Crisis

     445 words 10% matched vs. 2061 words 2% matched We invented medicines to kill bacteria; now, the bacteria are killing us. Lucien Swetschinski, a researcher and co-author at IHME, told…

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    Study: The Effect of Patient Support Groups on Pulmonary Hypertension Patients and their Caregivers
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    Study: The Effect of Patient Support Groups on Pulmonary Hypertension Patients and their Caregivers

    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…

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    IDEAYA’s IDE161 Receives Fast Track Designation for HR+ Her2- Breast Cancer
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    IDEAYA’s IDE161 Receives Fast Track Designation for HR+ Her2- Breast Cancer

      The FDA has granted IDEAYA Biosciences fast track status for IDE161 as a treatment for HR+, Her2- breast cancer. The therapy is a selective inhibitor of ADP ribose glycohydrolase…

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    November was Alzheimer’s Awareness Month
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    November was Alzheimer’s Awareness Month

      The need for increased awareness about Alzheimer’s disease is growing. The number of Alzheimer’s patients has increased since 1983, the year President Reagan designated November as Alzheimer’s Disease Awareness…

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    U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell
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    U.K. Health Authorities Grant Approval for a New Medicine using CRISPR Gene Editing to Treat Beta thalassemia and Sickle Cell

    CRISPR was first described ten years ago as a viral mechanism for defense, taken from bacteria, which could be transformed into genetic scissors and accurately alter DNA. About Casgevy CRISPR…

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    Phase III Trial meets Primary Endpoint for Patients with Eosinophilic Granulomatosis with Polyangiitis (EGPA)
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    Phase III Trial meets Primary Endpoint for Patients with Eosinophilic Granulomatosis with Polyangiitis (EGPA)

      Patients with severe asthma with an eosinophilic phenotype who are 12 years of age or older may now receive treatment with FASENRA as an add-on maintenance treatment. Note that…

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    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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