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.

    Artificial Intelligence(AI) and a Solution to Detect Familial Hypercholesterolemia

      Familial Hypercholesterolemia (FH) is often misdiagnosed as plain old high cholesterol because of overlapping symptoms such as elevated lipid levels. According to a recent article in MedicalView, FH is three…

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    New England Journal of Medicine (NEJM) Announces Results of A Unique Study of Hirschsprung Disease Affecting Infants

      An article in BioSpace reports new information published online by the NEJM concerning Hirschsprung disease. The disease involves the absence of nerves in parts of the intestines prior to birth. Researchers…

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    The First Adeno-Associated Viral Gene Therapy for Huntington’s Disease Receives the FDA’s Fast Track Designation
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    The First Adeno-Associated Viral Gene Therapy for Huntington’s Disease Receives the FDA’s Fast Track Designation

      A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…

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    uniQure Receives FDA Fast Track Designation for AMT 130 Gene Therapy for the Treatment of Huntingtons Disease

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands - uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs,  announced that the U.S. Food and Drug…

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    New Drug Application on Track for LCFAOD A Rare Autosomal Recessive Disease

    A recent article in the publication CheckOrphan announced encouraging results of a long term extension study sponsored by Ultragenyx involving UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorders…

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    Researchers in Sweden Studied 116,330 Twins and This is What They Discovered About Autoimmune Diseases

      According to a recent article from EurekAlert!, researchers used data from the Swedish Twin Registry and found that most of the 7 diseases they were studying were inherited.  The second finding…

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    An International Organization Dedicated to  Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals

    Three cases of fibrodysplasia ossificans progressiva (FOP) a rare genetic disease, were reported recently in Stat’s health issue. Wendy’s Story Wendy Weldon was a brave little nine years old when she…

    Continue Reading An International Organization Dedicated to  Finding a Cure for Fibrodysplasia Ossificans Progressiva (FOP) Began with a Group of Eleven Concerned Pen Pals

    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…

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    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…

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    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)