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 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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    Venclyxto® Plus Obinutuzumab Offers Previously Untreated CLL Patients Chemotherapy-Free Treatment
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    Venclyxto® Plus Obinutuzumab Offers Previously Untreated CLL Patients Chemotherapy-Free Treatment

      A recent press release by the global biopharmaceutical research company, AbbVie, reported a positive opinion by CHMP granted to VENCLYXTO® combined with obinutuzumab to treat chronic lymphocytic leukemia (CLL). A positive opinion by…

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    With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

    The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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    Biogen to Relaunch its Alzheimer’s Drug with FDA Clearance in New Study
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    Biogen to Relaunch its Alzheimer’s Drug with FDA Clearance in New Study

      According to an article in the Alzheimer’s publication Being Patient, participants who had been enrolled in the discontinued phase three trials of aducanumab will start receiving the drug as…

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    ICYMI: Johnson & Johnson and the UK Government: Genomics For Disease Prevention
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    ICYMI: Johnson & Johnson and the UK Government: Genomics For Disease Prevention

      Janssen R&D, a division of Johnson & Johnson (J&J), has partnered with the UK Biobank and others according to a recent news release by 3BL Media. It is noteworthy…

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    This Glomerulonephritis Patient has Been Receiving Dialysis Treatment for Over 30 Years. Now he Shares his Discipline with Other Patients
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    This Glomerulonephritis Patient has Been Receiving Dialysis Treatment for Over 30 Years. Now he Shares his Discipline with Other Patients

      According to a recent article in the MalayMail, it occurred to Abdullah’s doctor that the 67-year-old would be an inspiration for others who have kidney failure. His nephrologist, Dr.…

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    Searching for the Cause of Lung Disease in Systemic Juvenile Idiopathic Arthritis
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    Searching for the Cause of Lung Disease in Systemic Juvenile Idiopathic Arthritis

      According to a recent article in MedPage Today’s 2019 year-end review, the number of life-threatening lung diseases in systemic juvenile idiopathic arthritis (sJIA) patients has been increasing. sJIA is…

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    Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1
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    Risdiplam Delivers An Improvement to Infants with Spinal Muscular Atrophy Type 1

    Genentech (Roche Group) announced positive results of its FIREFISH trial in a recent Business Wire news release.  The second part of the study evaluated risdiplam’s efficacy on infants ages one…

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    A New University of Alabama Study Finds That Norepinephrine is the Missing Piece in the Alzheimer’s Puzzle
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    A New University of Alabama Study Finds That Norepinephrine is the Missing Piece in the Alzheimer’s Puzzle

      It has been evident to scientists for years that Alzheimer’s disease, which affects about five million people in the U.S., is caused by an accumulation of two proteins within…

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    FDA’s Acceptance of AYVAKIT’s New Drug Application Offers Hope to Metastatic GIST Patients
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    FDA’s Acceptance of AYVAKIT’s New Drug Application Offers Hope to Metastatic GIST Patients

      Surgery has been found to often be the best option towards a cure for a cancerous tumor (resectable) but there are many other tumors that are diagnosed as unresectable…

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    FDA Grants Orphan Drug Designation to Cellectar’s CLR 131 for the Sixth Time

      The biopharmaceutical company Cellectar Biosciences recently announced that the FDA granted its lead phospholipid drug conjugate, CLR 131, Orphan Drug Designation(ODD) for treatment of lymphoplasmacytic lymphoma. ODD is granted by…

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    ICYMI: A New Year, a New Autoinflammatory Disease, and a Promising New Drug
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    ICYMI: A New Year, a New Autoinflammatory Disease, and a Promising New Drug

      According to a recent article in Pharma Tutor, researchers at NHGRI, a division of the National Institute of Health, identified the biological cause of a newly-discovered autoinflammatory disease they named…

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