News

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 notable…

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…

Cell therapies may be a feasible way of achieving a long-term correction of bleeding disorders, preclinical data shared by Sigilon Therapeutics show. A single implant of Sigilon’s engineered cells given to mice enabled durable production of therapeutic levels of blood clotting factors and normalized bleeding time. The technology uses the…

Investigational gene therapy SB-525 can safely induce durable clotting factor VIII (FVIII) activity in patients with severe hemophilia A, preliminary data from the Phase 1/2 Alta clinical trial show. The trial’s most recent results were discussed at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress in…

Recommendations for vaccination practices for patients with hemophilia should be reassessed in an effort to minimize fear and emphasize the importance of vaccination to prevent infections, suggests a survey among physicians in Germany. Data from the PEDNET registry, a database of children with hemophilia, suggest that vaccination in close…

It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…