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March 20, 2019March 20, 2019

New Biomarker May Help Predict Response to Preventive Treatment with Factor VIII, Study Shows

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New evidence suggests that a parameter called FVIII-EC50 has potential as a new way to predict the response of an individual with severe hemophilia to preventive treatment with FVIII, ... Read more

March 18, 2019March 18, 2019

Prophylaxis Partially Prevents Bleeding in Severe Hemophilia A Patients, Real‐world Study Finds

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Regular preventive treatment is able to at least partially prevent bleeding episodes in adolescent and adult males with severe hemophilia A and without inhibitors, according to a real-world study. The ... Read more

March 15, 2019March 15, 2019

Hemlibra Approved in Europe to Prevent Bleeds in Hemophilia A Patients Without Factor VIII Inhibitors

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People with severe hemophilia A living in Europe can now receive treatment with Hemlibra (emicizumab-kxwh) to prevent bleeding episodes. The European Commission approved the prophylactic use of Hemlibra in ... Read more

March 14, 2019March 14, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

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Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of ... Read more

March 13, 2019March 13, 2019

Many Adults, Families Affected by Hemophilia Support Preconception, Prenatal Genetic Screens, Study Finds

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More than half of adults who either have hemophilia or are family members affected by the disease support the use of preconception and prenatal genetic screens, a survey reveals. ... Read more

March 13, 2019March 13, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

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With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed ... Read more

March 11, 2019March 11, 2019

Erectile Dysfunction More Prevalent with Age in Men With Hemophilia, Study Says

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Erectile dysfunction, more commonly known as male impotence, is associated with older age in men with hemophilia, especially those who smoked long term and have high blood pressure, a study ... Read more

March 8, 2019March 8, 2019

Advate is Safe, Effective Therapy for Hemophilia A, Japanese Study Finds

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Advate is an effective and well-tolerated treatment among patients with hemophilia A, according to an observational, long-term, Japanese study. The treatment showed better results when used as a preventive, rather than ... Read more

March 6, 2019March 6, 2019

Different Treatment Strategies May Affect Sports Participation in Hemophilia Patients, Study Suggests

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Hemophilia patients who take intermediate‐dose preventive medication may experience more of an age-related decline in sports participation, joint status, and physical functioning than those on a high dose, a ... Read more

March 6, 2019March 6, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

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Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known ... Read more

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