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August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

News

Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

July 31, 2019July 31, 2019

Hemlibra May Surpass Bypassing Agents in AHA, Study Says

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An 87-year old woman with acquired hemophilia A (AHA) showed no adverse events or major bleeding events after starting on Hemlibra (emicizumab). The findings suggest that Hemlibra may be ... Read more

July 29, 2019July 29, 2019

BioMarin Preparing to Submit Marketing Authorization Applications for Valoctocogene Roxaparvovec

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BioMarin Pharmaceuticals is planning to submit marketing authorization applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) seeking approval of valoctocogene roxaparvovec, its ... Read more

July 26, 2019July 26, 2019

Gene Editing Restores Factor VIII Levels, Eases Bleeding in Mice with Hemophilia A

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Combining two gene editing approaches in cells derived from a patient with severe hemophilia A successfully restored the levels and activity of the faulty clotting factor VIII (FVIII) and ... Read more

July 24, 2019July 24, 2019

Bayer Recalls Mislabeled Kogenate Vials Distributed in the U.S.

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Bayer is recalling two lots of product vials that have been wrongly labeled as Kogenate FS antihemophilic factor (recombinant) 2000 IU, when in reality they contain Jivi antihemophilic factor ... Read more

July 22, 2019July 22, 2019

Longer-lasting Jivi Improves Health and Life Quality, Men with Severe Hemophilia A Say

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Less frequent infusions with Jivi led to fewer bleedings, a greater ability to engage in physical and workplace activities, less vein damage, and better emotional well-being in men with severe ... Read more

July 19, 2019

European Initiative Targets Diagnosis, Treatment of Rare Diseases

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A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of ... Read more

July 19, 2019July 19, 2019

Two-stage Gene Therapy for Hemophilia A Shows Promising Results in Mice, Researchers Say

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Casebia’s two-stage gene therapy approach was successful in delivering the human gene coding for clotting factor VIII (FVIII) in a mouse model of hemophilia A, leading to stable and ... Read more

July 17, 2019July 17, 2019

New Data Support Long-term Effectiveness of Preventive EHL Treatment in Hemophilia A and B

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New data from clinical studies suggests that long-term preventive treatment of severe hemophilia A with Eloctate, or severe hemophilia B with Alprolix, increased quality of life and reduced annual bleeding ... Read more

July 15, 2019July 15, 2019

Esperoct Approved in Canada for Treatment of Hemophilia A

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Health Canada has approved Esperoct, an engineered formulation of clotting factor VIII, for the treatment of hemophilia A in people of all ages. The approval covers prophylactic, or preventive ... Read more

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