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.

    Parkinson’s: Newly Approved Drug Gives Advanced-Stage Patients Longer Periods of Relief

    The FDA’s recent approval of AbbVie’s new combination therapy provides patients with longer-lasting control of movements. As described recently in Biopharmadive, Parkinson’s disease is characterized by uncontrolled muscle movements or…

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    Gene Therapy Promising for Cerebral Adrenoleukodystrophy but Carries Risk of Blood Cancer
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    Gene Therapy Promising for Cerebral Adrenoleukodystrophy but Carries Risk of Blood Cancer

    The use of the gene therapy elivaldogene autotemcel (Skysona), has been approved due to its success in the treatment of patients diagnosed with the neurological disease cerebral adrenoleukodystrophy (CALD). The…

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    Positive Trial Data is Welcomed News for Spinal Muscular Atrophy Patients

    Spinal muscular atrophy (SMA) is a rare disease causing progressive muscular weakness. Severe forms of the disease can be fatal to infants. Apitegromab is an experimental therapy that was successful…

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    It Took Almost 60 Years for the Correct Diagnosis and Treatment for Primary Immunodeficiency
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    It Took Almost 60 Years for the Correct Diagnosis and Treatment for Primary Immunodeficiency

    Eighty-five-year-old Frank Meuers considers his 500th immunoglobulin infusion to be a cause for celebration. Frank has logged details of his doctor’s visits since his first dose of replacement therapy with…

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    Alzheimer’s: Researchers at Japan’s Riken Center Discover a New Treatment to Alleviate Symptoms and Improve Memory
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    Alzheimer’s: Researchers at Japan’s Riken Center Discover a New Treatment to Alleviate Symptoms and Improve Memory

    The Riken Center team, headed by Dr. Takaomi Saido and using mice with Alzheimer’s, discovered that dopamine may lessen the brain’s physical symptoms and improve memory. The study, featured in…

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    ‘Anyone with a Heart’ Campaign Spreads Awareness About Hereditary ATTR Amyloidosis
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    ‘Anyone with a Heart’ Campaign Spreads Awareness About Hereditary ATTR Amyloidosis

    Fredericksburg, Virginia resident Johnny Boatman is a former national guardsman who had been in excellent physical condition all his life. That was until 2022 when he felt a sharp pain…

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    Blood Tests May be the Future for Early Alzheimer’s Detection
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    Blood Tests May be the Future for Early Alzheimer’s Detection

    It is estimated that over six million people in the U.S. have Alzheimer’s disease plus millions of other people throughout the world. Alzheimer’s is the most common type of dementia.…

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    Small Study Finds Large Benefits for People With Friedreich’s Ataxia Heart Complications
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    Small Study Finds Large Benefits for People With Friedreich’s Ataxia Heart Complications

    A recent article at BioPharma Dive outlines the findings of a study into Lexeo’s experimental gene therapy. The six participants in the recently conducted interim trial had enlarged hearts associated…

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    Study: 77% of Pregnancies Result in Live Births After Stem Cell Transplant
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    Study: 77% of Pregnancies Result in Live Births After Stem Cell Transplant

    Women who undergo allogeneic hematopoietic cell transplantation (alloHCT) face significant treatment-related challenges regarding fertility. However, science seems to be overcoming many treatment-related fertility challenges after alloHCT and successful pregnancies are…

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    The First Successful Attempt to Study Alzheimer’s Disease Using Lab-Derived Neurons
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    The First Successful Attempt to Study Alzheimer’s Disease Using Lab-Derived Neurons

    Researchers at Washington University’s medical school have used a new technique to study brain neurons in a lab setting, allowing them to bypass the need to rely on a brain…

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    Positive Results from the Phase 3 Trial of ANX005 in Guillain-Barré Syndrome
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    Positive Results from the Phase 3 Trial of ANX005 in Guillain-Barré Syndrome

    The long-awaited positive results of Annexon Inc.’s Phase 3 trial investigating ANX005 for Guillain-Barré syndrome (GBS) was presented at the PNS 2024 Annual Meeting. Several leading global GBS experts spoke…

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