Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

    GAPVAC Project Tests An Individualized Form of  Immunotherapy to Treat Aggressive Brain Cancer

    A recent article in the journal Nature reports that Phase 1 GAPVAC-101 First-In-Human trial is one of the first clinical trials with a goal of designing and manufacturing actively personalized vaccines…

    Continue Reading GAPVAC Project Tests An Individualized Form of  Immunotherapy to Treat Aggressive Brain Cancer

    Secondary Long-Term Prophylaxis Shows Promise for Patients with Severe von Willebrand Disease (vWD)

    A recent article in the National Hemophilia Foundation news quotes the Blood Transfusion journal's report on the results of a study conducted under the lead of Dr. Flora Peyvandi, Medical…

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    A Record Number of FDA Drug Approvals in 2018 Is Followed by an Encouraging Start for 2019

    Following a record number of approvals in 2018, the FDA has recently approved two new drugs with many more anticipated approvals later in the year. Trazimera to Treat Human Epidermal…

    Continue Reading A Record Number of FDA Drug Approvals in 2018 Is Followed by an Encouraging Start for 2019

    International Genetic Research Group to Begin Study of Hypermobile Ehlers-Danlos Syndrome (hEDS)

    A recent announcement by the Ehlers-Danlos Society marks the beginning of the recruitment phase for an international study devoted to determining the root cause of hEDS. The gathering of this…

    Continue Reading International Genetic Research Group to Begin Study of Hypermobile Ehlers-Danlos Syndrome (hEDS)
    MD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer
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    MD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer

    Although some ultrasound and/or CT scan studies may show that patients’ lymph nodes (glands) are free of disease, upon further examination using sentinel lymph node biopsies,(SLNB) the doctors at MD…

    Continue Reading MD Anderson Physicians Use a Diagnostic Method Called Sentinel Lymph Node Biopsy To Determine Spread of Cancer

    FDA Regulations and the Critical Differences Between Medical Device Trials and Pharmaceutical Trials

    Medical devices include ECG electrodes, bandages, cardiac pacemakers, thermometers, and hemodialysis machines. A comparison of clinical trials shows that medical device trials are somewhat similar to pharmaceutical trials that evaluate drugs,…

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    Scientists are Ramping Up Their Attack on Alzheimer’s Disease Through Immunotherapy

    In the early 1900s, German psychiatrist Alois Alzheimer, while studying about the decline in brain function, recognized a correlation between behavioral changes and the buildup of tangles and plaques in…

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    Rare Diseases Could Be Diagnosed Earlier with Better Training for GPs to Avoid Years of Misdiagnosis

    Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…

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    Researchers Fail to Find a Cure for MPS Through Genome Editing

    The Daily News recently published an announcement by researchers at Sangamo Therapeutics showing the results of its first human trial to treat two rare genetic disorders through gene-editing technology. Gene-editing (or…

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    Vancouver Researchers Find That Blood Cells Could Help Screen Ambroxol’s Efficacy in GD Type 3

      A recent article in CheckOrphan describes a study that was conducted by Researchers at the Lysosomal and Rare Disorders Research and Treatment Center in Vancouver, B.C. The study examines the…

    Continue Reading Vancouver Researchers Find That Blood Cells Could Help Screen Ambroxol’s Efficacy in GD Type 3

    Alexion’s Ultomiris™ Met the Primary Objective in a Phase 3 Trial of Atypical Hemolytic Uremic Syndrome Patients

    Alexion Pharmaceuticals, Inc., a company that is advancing a rare disease pipeline in complement biology, recently issued a series of press releases announcing positive results of a Phase 3 study…

    Continue Reading Alexion’s Ultomiris™ Met the Primary Objective in a Phase 3 Trial of Atypical Hemolytic Uremic Syndrome Patients

    Researchers Find the Future of Ofatumumab in Treating Follicular Lymphoma is Questionable

    Ofatumumab as a Single Agent  Results of ofatumumab administered as a single agent with an extended induction (initial treatment) brought about a high response rate and it was well tolerated among follicular…

    Continue Reading Researchers Find the Future of Ofatumumab in Treating Follicular Lymphoma is Questionable

    Merck’s Keytruda in Combination with Pfizer’s Inlyta Significantly Improved Overall Survival and Progression-Free Survival in Therapy for Metastatic or Advanced Renal Cell Carcinoma

      The treatment of metastatic renal cell carcinoma (RCC) has evolved over the last ten years and investigators now look towards combination therapies to improve response rates and durability for…

    Continue Reading Merck’s Keytruda in Combination with Pfizer’s Inlyta Significantly Improved Overall Survival and Progression-Free Survival in Therapy for Metastatic or Advanced Renal Cell Carcinoma

    FDA Approves Venetoclax Combination For Newly Diagnosed AML in Adults 75 Years or Older

    The Food and Drug Administration (FDA) recently announced its approval of Venetoclax (Venclexta)  in combination with either decitabine or azacitidine (hypomethylating agents) or cytarabine (an antimetabolite) to treat adults 75 years…

    Continue Reading FDA Approves Venetoclax Combination For Newly Diagnosed AML in Adults 75 Years or Older

    University of Louisville’s Research Team Discovers Molecule That Protects Against Development of Cancer Cells

    The University of Louisville’s publication UL News recently reported a finding by its research team regarding an immune checkpoint molecule.  The molecule had been developed for cancer immunotherapy but when…

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