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 today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…
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 a better option for bleeding prevention in AHA patients than bypassing agents, the team said. The study, “…
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 investigational gene therapy for the treatment of adults with hemophilia A, before the end of…
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 reduced bleedings in a mouse model of the disease, according to a new study. The research, “ssODN-Mediated…
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 (recombinant) PEGylated-aucl 3000 IU. Both Kogenate (BAY14-2222) and Jivi (BAY 94-9027) are factor VIII replacement therapies…
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 hemophilia A compared to previous therapies used, participants in a Phase 2/3 trial largely agreed. “Exploring the Impact of…
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 treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…
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 increasing levels of the factor, researchers said. “Our scientific team, led by Alan Brooks, Ph.D., have achieved a…
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 rates. The data was presented in Melbourne, Australia at ISTH 2019, the 27th Congress of the International Society…
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 treatment, on-demand use for controlling bleeding episodes, and use in reducing bleeding during surgery. Esperoct received similar approval…
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