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.

    Antiphospholipid Syndrome (APS): Young Women are Five Times More Likely Than Men to Have This Rare Disease
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    Antiphospholipid Syndrome (APS): Young Women are Five Times More Likely Than Men to Have This Rare Disease

    According to a recent article in Healio, the National Institute of Health states that one-third of strokes occurring for the first time to people under fifty can be linked to…

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    Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders
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    Clinical Trial of MGTA-456 Looks Promising for Inherited Metabolic Disorders

      According to an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…

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    ICYMI: She was Diagnosed with Hashimoto’s Thyroiditis but her Doctor Didn’t Know How to Treat It
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    ICYMI: She was Diagnosed with Hashimoto’s Thyroiditis but her Doctor Didn’t Know How to Treat It

      Susan Mann was seven years old when she was diagnosed as having Hashimoto’s thyroiditis, an autoimmune disease that affects the thyroid. According to a recent article in the online…

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    Mitochondrial Disease Patient’s Parents Lose Law Suit Against Four Doctors and Boston Children’s Hospital
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    Mitochondrial Disease Patient’s Parents Lose Law Suit Against Four Doctors and Boston Children’s Hospital

      The Boston Globe recently interviewed the principals in a case brought by the Pelletier family against the Boston Children’s Hospital. The article gives details about a family’s charge of…

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    Neurodegenerative-Neuromuscular Diseases and Airway Clearance Therapy
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    Neurodegenerative-Neuromuscular Diseases and Airway Clearance Therapy

      Advances in medicine have been responsible for longer overall survival in patients with neurodegenerative-neuromuscular diseases. According to a recent report in RTMagazine, an increase in the number of patients…

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    This College Student Turned her Spastic Diplegic Cerebral Palsy into Projects for the Disabled
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    This College Student Turned her Spastic Diplegic Cerebral Palsy into Projects for the Disabled

      Jennifer Schlegel’s life began earlier than expected with an unanticipated disease. According to an article in The Lantern, Jennifer was born prematurely and diagnosed with spastic diplegic cerebral palsy.…

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    The EMA Grants PRIME Designation to Viralym-M To Treat Viral Infections Linked to Stem Cell Transplant

      According to a recent article in The Pharma Letter, the European Medicines Agency (EMA) granted PRIME (PRIority MEdicines) designation to Viralym-M based on positive results from a Phase 2 proof-of-concept…

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    Toxic Protein Identified: Closing in on Alzheimer’s and Neurodegenerative Diseases
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    Toxic Protein Identified: Closing in on Alzheimer’s and Neurodegenerative Diseases

      For years scientists have tried to understand how and why tau, well known to researchers for its implication in Alzheimer’s disease, changes from its normal form to a harmful…

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    Pancreatic Cancer: Researchers are Calling Patients’ Response to this Treatment “Unprecedented“
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    Pancreatic Cancer: Researchers are Calling Patients’ Response to this Treatment “Unprecedented“

      The ASCO Post recently reported findings of high response rates and improved survival from the combination of platinum-based cisplatin and gemcitabine (chemotherapy) in BRCA/PALB2 mutation positive pancreatic ductal adenocarcinoma. The goal…

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    The FDA is Developing, Approving, and Expediting Exciting New Therapies
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    The FDA is Developing, Approving, and Expediting Exciting New Therapies

      Janet Woodcock, Director of the FDA’s Center for Drug Evaluation and Research (CDER) recently gave an interview to the publication FDA Voices about the agency’s approval in 2019 of a…

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    An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit
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    An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit

      On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…

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    The Halycon Machine Could Mark a Major Improvement in Radiation Treatment
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    The Halycon Machine Could Mark a Major Improvement in Radiation Treatment

      The Halcyon machine holds an imaging mechanism that produces a high-quality CAT scan plus it houses a treatment unit that delivers radiation. According to a recent news release, approximately twenty-four…

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