Results 31 to 40 of about 12,566,431 (163)

APTAMERS ARE NEW PHARMACOLOGICAL SUBSTANCES FOR THE DEVELOPMENT OF ANTICOAGULANTS

Атеротромбоз, 2017
A ptamers are a new class of oligonucleotide compounds capable of specific binding to various molecular targets and inhibiting their activity. Aptamers are selected from a library of randomly syn-thesized oligonucleotides (from 20 to 60 nucleotides long)
A. V. Mazurov, V. A. Spiridonova
doaj   +1 more source

Gene Therapy and Hemophilia: Where Do We Go from Here?

Journal of Blood Medicine, 2022
Nancy S Bolous,1 Nidhi Bhatt,2 Nickhill Bhakta,1 Ellis J Neufeld,2 Andrew M Davidoff,3 Ulrike M Reiss2 1Department of Global Pediatric Medicine – St.
Bolous NS, Bhatt N, Bhakta N, Neufeld EJ, Davidoff AM, Reiss UM   +5 more
doaj  

RENAL ALLOGENEIC TRANSPLANTATION IN PATIENT WITH HAEMOPHILIA B

Вестник трансплантологии и искусственных органов, 2014
We report the case of successful renal allogeneic transplantation and treatment in a 56-year-old patient with haemophilia B at Hematology Research Center. He has received replacement therapy by factor IX since 2010.
N. V. Purlo, R. B. Achmetshin, A. J. Bulanov, E. A. Likhacheva, L. S. Birjukova   +4 more
doaj   +1 more source

Cochlear implantation in hemophilia B—a rare case report

The Egyptian Journal of Otolaryngology, 2022
Background Hemophilia B is an X-linked inherited disease, mainly caused by deficiency of factor IX. Severity of the disease is manifested by the factor IX deficiency in the blood.
Chetan Yadav, Diva Sharma, Neeraj Suri, Meenesh Juvekar   +3 more
doaj   +1 more source

Treatment of hemophilia B: focus on recombinant factor IX

Biologics : targets & therapy, 2013
Hemophilia B is a recessive X-linked bleeding disorder characterized by deficiency of the coagulation factor IX (FIX). In hemophilia B patients the severity of the bleeding phenotype is related to the degree of the FIX defect.
M. Franchini, F. Frattini, S. Crestani, C. Sissa, C. Bonfanti   +4 more
semanticscholar   +1 more source

Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs

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, Ying Poi Liu, Jacek Lubelski, Erich Ehlert, Sander Gielen, Paula Montenegro-Miranda, Martin de Haan, Bart Nijmeijer, Valerie Ferreira, Harald Petry, Sander J. van Deventer   +10 more
doaj  

Real-World Amount of Clotting Factor Concentrates Dispensed and Annual Medical Expenditures for Japanese Patients with Hemophilia B

Journal of Blood Medicine, 2023
Katsuyuki Fukutake,1,2 Kanae Togo,3 Linghua Xu,3 Leona E Markson,4 José Maria Jimenez Alvir,5 Ian Winburn,6 Toshiyuki Karumori3 1Laboratory Medicine, Tokyo Medical University, Shinjuku, Tokyo, Japan; 2Department of Blood Coagulation Diseases, Ogikubo ...
Fukutake K, Togo K, Xu L, Markson LE, Alvir JMJ, Winburn I, Karumori T   +6 more
doaj  

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

EMBO Molecular Medicine, 2013
Formation of pathogenic antibodies is a major problem in replacement therapies for inherited protein deficiencies. For example, antibodies to coagulation factors (‘inhibitors’) seriously complicate treatment of haemophilia.
D. Markusic, Brad E. Hoffman, G. Perrin, Sushrusha Nayak, Xiaomei Wang, Paul A. LoDuca, K. High, R. Herzog   +7 more
semanticscholar   +1 more source

Signaling mechanisms of the platelet glycoprotein Ib-IX complex

Platelets, 2022
The glycoprotein Ib–IX (GPIb-IX) complex mediates initial platelet adhesion to von Willebrand factor (VWF) immobilized on subendothelial matrix and endothelial surfaces, and transmits VWF binding-induced signals to stimulate platelet activation.
Yaping Zhang, Samuel M Ehrlich, Cheng Zhu, Xiaoping Du   +3 more
doaj   +1 more source

Activation of factor IX by the reaction product of tissue factor and factor VII: additional pathway for initiating blood coagulation.

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, Samuel l. Rapaport, Bjarne 0sterud   +2 more
semanticscholar   +1 more source