Literature DB >> 22389200

The hope and reality of long-acting hemophilia products.

Steven W Pipe1.   

Abstract

Recombinant DNA technology and protein engineering are creating hope that we can address ongoing challenges in hemophilia care such as reducing the costs of therapy, increasing the availability to the developing world, and improving the functional properties of these proteins. Technological advances to improve the half-life of recombinant clotting factors have brought long-acting clotting factors for hemophilia replacement therapy closer to reality. Preclinical and clinical trial results are reviewed as well as the potential benefits and risks of these novel therapies.
Copyright © 2012 Wiley Periodicals, Inc.

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Year:  2012        PMID: 22389200     DOI: 10.1002/ajh.23146

Source DB:  PubMed          Journal:  Am J Hematol        ISSN: 0361-8609            Impact factor:   10.047


  14 in total

Review 1.  Present and future challenges in the treatment of haemophilia: the patient's perspective.

Authors:  Romano Arcieri; Angelo C Molinari; Stefania Farace; Giuseppe Mazza; Alberto Garnero; Gabriele Calizzani; Paola Giordano; Emily Oliovecchio; Lorenzo Mantovani; Lamberto Manzoli; Paul Giangrande
Journal:  Blood Transfus       Date:  2013-09       Impact factor: 3.443

Review 2.  Present and future challanges in the treatment of haemophilia: a clinician's perspective.

Authors:  Pier Mannuccio Mannucci; Massimo Franchini
Journal:  Blood Transfus       Date:  2013-09       Impact factor: 3.443

3.  A von Willebrand factor fragment containing the D'D3 domains is sufficient to stabilize coagulation factor VIII in mice.

Authors:  Andrew Yee; Robert D Gildersleeve; Shufang Gu; Colin A Kretz; Beth M McGee; Keisha M Carr; Steven W Pipe; David Ginsburg
Journal:  Blood       Date:  2014-05-21       Impact factor: 22.113

4.  BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice.

Authors:  Ekta Seth Chhabra; Tongyao Liu; John Kulman; Susannah Patarroyo-White; Buyue Yang; Qi Lu; Douglas Drager; Nancy Moore; Jiayun Liu; Amy M Holthaus; Jurg M Sommer; Ayman Ismail; Deana Rabinovich; Zhan Liu; Arjan van der Flier; Allison Goodman; Chris Furcht; Mark Tie; Tyler Carlage; Randy Mauldin; Terrence M Dobrowsky; Zhiqian Liu; Oblaise Mercury; Lily Zhu; Baisong Mei; Volker Schellenberger; Haiyan Jiang; Glenn F Pierce; Joe Salas; Robert Peters
Journal:  Blood       Date:  2020-04-23       Impact factor: 22.113

5.  Tailoring of medical treatment: hemostasis and thrombosis towards precision medicine.

Authors:  Giovanni Di Minno; Elena Tremoli
Journal:  Haematologica       Date:  2017-02-28       Impact factor: 9.941

6.  Anti-factor IXa/X bispecific antibody ACE910 prevents joint bleeds in a long-term primate model of acquired hemophilia A.

Authors:  Atsushi Muto; Kazutaka Yoshihashi; Minako Takeda; Takehisa Kitazawa; Tetsuhiro Soeda; Tomoyuki Igawa; Zenjiro Sampei; Taichi Kuramochi; Akihisa Sakamoto; Kenta Haraya; Kenji Adachi; Yoshiki Kawabe; Keiji Nogami; Midori Shima; Kunihiro Hattori
Journal:  Blood       Date:  2014-10-01       Impact factor: 22.113

7.  Hemophilia in Sports: A Case Report and Prophylactic Protocol.

Authors:  Mark Maffet; Jimmy Roton
Journal:  J Athl Train       Date:  2016-11-18       Impact factor: 2.860

Review 8.  Progress towards gene therapy for haemophilia B.

Authors:  Nishil Patel; Ulrike Reiss; Andrew M Davidoff; Amit C Nathwani
Journal:  Int J Hematol       Date:  2014-02-06       Impact factor: 2.490

9.  Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Authors:  Amit C Nathwani; Ulreke M Reiss; Edward G D Tuddenham; Cecilia Rosales; Pratima Chowdary; Jenny McIntosh; Marco Della Peruta; Elsa Lheriteau; Nishal Patel; Deepak Raj; Anne Riddell; Jun Pie; Savita Rangarajan; David Bevan; Michael Recht; Yu-Min Shen; Kathleen G Halka; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; James Allay; Mark A Kay; Catherine Y C Ng; Junfang Zhou; Maria Cancio; Christopher L Morton; John T Gray; Deokumar Srivastava; Arthur W Nienhuis; Andrew M Davidoff
Journal:  N Engl J Med       Date:  2014-11-20       Impact factor: 176.079

10.  Health-Related Quality of Life in Children and Adolescents with Hereditary Bleeding Disorders and in Children and Adolescents with Stroke: Cross-Sectional Comparison to Siblings and Peers.

Authors:  Bruno Neuner; Sylvia von Mackensen; Susanne Holzhauer; Stephanie Funk; Robert Klamroth; Karin Kurnik; Anne Krümpel; Susan Halimeh; Sarah Reinke; Michael Frühwald; Ulrike Nowak-Göttl
Journal:  Biomed Res Int       Date:  2016-05-15       Impact factor: 3.411

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