ALLMedicine™ Hemophilia B Center

Mar 4th, 2021 - In the rare co-occurrence of childhood cancer and severe hemophilia, hemostatic management is of paramount therapeutic importance. We present the case of an 11-month-old boy with severe congenital hemophilia B, who was diagnosed with metastatic hi...

Mar 4th, 2021 - Stem cell transplantation to the liver is a promising therapeutic strategy for a variety of disorders. Hepatocyte transplantation has short-term efficacy but can be problematic due to portal hypertension, inflammation, and sinusoidal thrombosis. W...

Mar 3rd, 2021 - The high-specific-activity factor IX (FIX) variant Padua (R338L) is the most promising transgene for hemophilia B (HB) gene therapy. Although R338 is strongly conserved in mammalian evolution, amino acid substitutions at this position are underrep...

Mar 1st, 2021 - Gene therapy may lead to a cure for hemophilia B (HB) if it is successful. Data from clinical trials using adeno-associated virus (AAV)-mediated liver-targeted FIX gene therapy are very encouraging. However, this protocol can be applied only to ad...

Feb 26th, 2021 - Limited information exists regarding the factor IX (FIX) coagulant activity (FIX:C) measured by different assays following FIX-Padua gene therapy. Assess for the first time FIX:C in 5 commonly used coagulation assays in plasma samples from hemophi...

Dec 4th, 2020 - Haemophilia B (HB) is less well studied than haemophilia A (HA); despite similarities between the two inherited bleeding disorders, important differences remain that require further research.

Nov 7th, 2020 - Hemophilia is the most common X‐linked bleeding disorder, affecting over 1 million individuals throughout the world. Patients are subclassified into mild, moderate and severe disease based on per cent factor activity level.

Oct 13th, 2020 - Hemophilia is an inherited disease that requires a different approach in order to evaluate, monitor and treat patients. Despite the great advances in therapeutic agents that have emerged, reports on the impact of monitoring outcomes on treatment d...

Aug 2nd, 2020 - This new edition of the World Federation of Hemophilia (WFH) guidelines for the management of hemophilia comes at an exciting time in the evolution of the diagnosis and treatment of this condition. Since the publication of the second edition in 20...

Jan 23rd, 2017 - Prior to the availability of clotting factor concentrates, the natural history of arthropathy in severe haemophilia was well described, with recurrent bleeds into joints leading to progressive joint damage and ultimate destruction with associa...

Mar 4th, 2021 - In the rare co-occurrence of childhood cancer and severe hemophilia, hemostatic management is of paramount therapeutic importance. We present the case of an 11-month-old boy with severe congenital hemophilia B, who was diagnosed with metastatic hi...

Mar 4th, 2021 - Stem cell transplantation to the liver is a promising therapeutic strategy for a variety of disorders. Hepatocyte transplantation has short-term efficacy but can be problematic due to portal hypertension, inflammation, and sinusoidal thrombosis. W...

Mar 3rd, 2021 - The high-specific-activity factor IX (FIX) variant Padua (R338L) is the most promising transgene for hemophilia B (HB) gene therapy. Although R338 is strongly conserved in mammalian evolution, amino acid substitutions at this position are underrep...

Mar 1st, 2021 - Gene therapy may lead to a cure for hemophilia B (HB) if it is successful. Data from clinical trials using adeno-associated virus (AAV)-mediated liver-targeted FIX gene therapy are very encouraging. However, this protocol can be applied only to ad...

Feb 26th, 2021 - Limited information exists regarding the factor IX (FIX) coagulant activity (FIX:C) measured by different assays following FIX-Padua gene therapy. Assess for the first time FIX:C in 5 commonly used coagulation assays in plasma samples from hemophi...

Dec 17th, 2020 - NEW YORK (Reuters Health) - An investigational gene therapy for hemophilia B substantially increased production of the blood-clotting protein factor IX and reduced the number of bleeding episodes and need for prophylactic therapy in a phase-3 tria...

Dec 4th, 2020 - Haemophilia B (HB) is less well studied than haemophilia A (HA); despite similarities between the two inherited bleeding disorders, important differences remain that require further research.

Nov 7th, 2020 - Hemophilia is the most common X‐linked bleeding disorder, affecting over 1 million individuals throughout the world. Patients are subclassified into mild, moderate and severe disease based on per cent factor activity level.

Oct 21st, 2020 - Hemophilia A is an inherited deficiency of FVIII and an X-linked recessive disorder. Hemophilia B, another recessive disorder, is characterized by a deficiency in factor IX. Hemophilia C is an autosomal recessive disorder in which patients are def...

Oct 13th, 2020 - Hemophilia is an inherited disease that requires a different approach in order to evaluate, monitor and treat patients. Despite the great advances in therapeutic agents that have emerged, reports on the impact of monitoring outcomes on treatment d...