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March 27, 2019March 27, 2019

UK Hemophilia Care Teams Lack Knowledge on Bleeding Risks in Dental Procedures, Study Suggests

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Hemophilia care providers in the U.K. have insufficient knowledge on how to assess the risk of prolonged bleeding associated with dental procedures, a study has found. The study, “U.K. ... Read more

March 25, 2019March 25, 2019

Population-Based Pharmacokinetic Dosing May Benefit Hemophilia A Patients, Study Reports

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A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The ... Read more

March 22, 2019March 22, 2019

Healthcare Costs Higher with Bypassing Agents than Factor Replacement Therapy in Hemophilia A, Study Reports

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Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare ... Read more

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

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