Successful immunosuppressive drug‐free immune tolerance induction in hemophilia B with inhibitor and anaphylaxis to factor IX: A case report [PDF]
Clinical Case ReportsKey Clinical Message Recommendations advise factor IX desensitization before immune tolerance induction in severe hemophilia B, supported by immunosuppression.
Ángeles Palomo Bravo +4 more
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The Clinical Genetics of Hemophilia B (Factor IX Deficiency) [PDF]
The Application of Clinical Genetics, 2021 Connie H Miller1,2 1Division of Blood Disorders, National Center on Birth Defects and Developmental Disabilities, Centers for Disease Control and Prevention, Atlanta, GA, USA; 2Synergy America, Inc., Duluth, GA, USACorrespondence: Connie H MillerDivision
Miller CH
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Proceedings of the National Academy of Sciences of the United States of America, 1977 A study was carried out on mechanisms, independent of activated Factor XI, capable of activating Factor IX. The reaction product of tissue factor and Factor VII functioned as a potent Factor IX activator in the assay system used.
Bjarne 0sterud +2 more
semanticscholar +2 more sources
Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs [PDF]
Molecular Therapy: Methods & Clinical Development, 2019 Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and safety of AMT-060, an investigational gene therapy comprising an adeno-associated virus serotype 5 ...
Elisabeth A. Spronck +10 more
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A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B [PDF]
Journal of Blood MedicineZoran Igrutinović,1,2 Hélène Louise Hooimeijer,3 Karim Kentouche,4 Jaco Botha,5 Peter L Turecek,6 Marta Kokot-Kierepa,5 Hanna T Gazda7 1Clinic of Pediatrics, Department of Hemato-Oncology, University Clinical Center of Kragujevac, Kragujevac ...
Igrutinović Z +6 more
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Factor IX p.A37V mutation causes severe bleeding in a patient with phenprocoumon therapy [PDF]
European Journal of Medical Research, 2021 Background Bleeding is the most common complication of oral anticoagulants, due to inadequate dosing. Case presentation This report describes the clinical course of a patient who developed severe bleeding under therapy with phenprocoumon, despite an INR ...
Nils Mülling +3 more
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CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs [PDF]
Haematologica, 2020 Hemophilia B is an X-linked recessive bleeding disorder caused by abnormalities in the coagulation factor IX gene. Without prophylactic treatment, patients experience frequent spontaneous bleeding episodes.
Jiahuan Chen +14 more
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Journal of Tissue Engineering, 2012 Cell microencapsulation holds significant promise as a strategy for cellular therapies; however, inadequate survival and functionality of the enclosed cells limit its application in hemophilia treatment.
Bahareh Sayyar +6 more
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Peri-operative Management and the Role of Minimally Invasive Spine Surgery in a Case of Hemophilia B [PDF]
Journal of Minimally Invasive Spine Surgery and Technique, 2022 Hemophilia A and B are rare X-chromosome-linked recessive bleeding disorders caused by mutations in the genes causing abnormalities of blood clotting factors VIII and IX, respectively.
VNR Praveen Goparaju +3 more
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