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About 2,563 results

ALLMedicine™ Hemophilia B Center

Research & Reviews  1,049 results

Hemostatic Management in an Infant With Neuroblastoma and Severe Hemophilia B With Exte...
https://doi.org/10.1097/MPH.0000000000002109
Journal of Pediatric Hematology/oncology; Cuntz F, Deubzer HE et. al.

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...

Endoderm and Hepatic Progenitor Cells Engraft in the Quiescent Liver Concurrent with In...
https://doi.org/10.1177/0963689721993780
Cell Transplantation; Fagg WS, Liu N et. al.

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...

Evolutionary insights into coagulation factor IX Padua and other high-specific-activity...
https://doi.org/10.1182/bloodadvances.2019000405
Blood Advances; Samelson-Jones BJ, Finn JD et. al.

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...

Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with pree...
https://doi.org/10.1182/bloodadvances.2020004071
Blood Advances; Schroeder JA, Chen J et. al.

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...

Factor IX Assay Discrepancies in the Setting of Liver Gene Therapy Using a Hyperfunctio...
https://doi.org/10.1111/jth.15281
Journal of Thrombosis and Haemostasis : JTH; Robinson MM, George LA et. al.

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...

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Guidelines  41 results

Natural history study of factor IX deficiency with focus on treatment and complications (B‐Natural)
https://onlinelibrary.wiley.com/doi/10.1111/hae.14139
Haemophilia Shapiro AD

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.

The role of total ankle replacement in patients with hemophilia and end‐stage ankle arthropathy: A review
https://onlinelibrary.wiley.com/doi/10.1111/hae.14196
Haemophilia Sackstein P

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.

Current practices in hemophilic patients undergoing orthopedic surgery - a systematic review
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7588779/
Experimental and Therapeutic Medicine; Badulescu OV

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...

WFH Guidelines for the Management of Hemophilia, 3rd edition
https://onlinelibrary.wiley.com/doi/10.1111/hae.14046
Haemophilia

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...

Guidelines for the management of acute joint bleeds and chronic synovitis in haemophilia: A United Kingdom Haemophilia Centre Doctors’ Organisation (UKHCDO) guideline
http://www.ukhcdo.org/wp-content/uploads/2017/03/Guidelines-for-the-management-of-acute-joint-bleeds-and-chronic-synovitis-in-haemophilia.pdf
Haemophilia

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...

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Drugs  31 results see all →

Clinicaltrials.gov  1,169 results

Hemostatic Management in an Infant With Neuroblastoma and Severe Hemophilia B With Exte...
https://doi.org/10.1097/MPH.0000000000002109
Journal of Pediatric Hematology/oncology; Cuntz F, Deubzer HE et. al.

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...

Endoderm and Hepatic Progenitor Cells Engraft in the Quiescent Liver Concurrent with In...
https://doi.org/10.1177/0963689721993780
Cell Transplantation; Fagg WS, Liu N et. al.

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...

Evolutionary insights into coagulation factor IX Padua and other high-specific-activity...
https://doi.org/10.1182/bloodadvances.2019000405
Blood Advances; Samelson-Jones BJ, Finn JD et. al.

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...

Platelet-targeted hyperfunctional FIX gene therapy for hemophilia B mice even with pree...
https://doi.org/10.1182/bloodadvances.2020004071
Blood Advances; Schroeder JA, Chen J et. al.

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...

Factor IX Assay Discrepancies in the Setting of Liver Gene Therapy Using a Hyperfunctio...
https://doi.org/10.1111/jth.15281
Journal of Thrombosis and Haemostasis : JTH; Robinson MM, George LA et. al.

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...

see more →

News  273 results

Gene Therapy a Potential 'Game Changer' for Hemophilia B
https://www.medscape.com/viewarticle/942762

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...

Natural history study of factor IX deficiency with focus on treatment and complications (B‐Natural)
https://onlinelibrary.wiley.com/doi/10.1111/hae.14139
Haemophilia Shapiro AD

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.

The role of total ankle replacement in patients with hemophilia and end‐stage ankle arthropathy: A review
https://onlinelibrary.wiley.com/doi/10.1111/hae.14196
Haemophilia Sackstein P

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.

Fast Five Quiz: Presentation and Diagnosis of Hemophilia A
https://reference.medscape.com/viewarticle/939102_2

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...

Current practices in hemophilic patients undergoing orthopedic surgery - a systematic review
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7588779/
Experimental and Therapeutic Medicine; Badulescu OV

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...

see more →