Results 31 to 40 of about 113,823 (359)

Perinatal Gene Transfer to the Liver [PDF]

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Chan, JK, Howe, SJ, McKay, TR, Rahim, AA, Waddington, SN, Ward, NJ   +6 more
core   +1 more source

A review of evidence about behavioural and psychological aspects of chronic joint pain among people with haemophilia [PDF]

, 2014
Joint pain related to haemophilia affects large numbers of people and has a significant impact on their quality of life. This article reviews evidence about behavioural and psychological aspects of joint pain in haemophilia, and considers that evidence ...
Acharya, Barlow, Barry, Buhrman, Buzzard, Cour, Eckman, Elander, Elander, Elander, Elander, Elander, Ersek, Fischer, Fischer, Fishbain, Galen, Genderen, Gil, Hermans, Hilberg, Holstein, Humphries, Ingerslev, Jensen, Jensen, Karimi, Korff, Laforest, LeFort, Lorig, Lorig, Lorig, Lusher, Mackensen, McCracken, McCracken, McCracken, McCracken, Moore, Moore, Mulvany, Neufeld, Niscola, O'Mahony, Penica, Poon, Riley, Risdon, Rodriguez-Merchan, Royal, Santavirta, Santavirta, Schramm, Schulz, Sherry, Solomon, Solovieva, Spitzer, Stover, Swirsky-Sacchetti, Szende, Thorn, Turk, Turk, Varni, Varni, Varni, Varni, Wallny, Wilson, Witkop, Witkop, Wright, Zawilska, Zhou   +75 more
core   +2 more sources

A long-term study of AAV gene therapy in hemophilia A dogs identifies clonal expansions of transduced liver cells

Nature Biotechnology, 2020
Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine factor VIII (AAV-cFVIII) corrected the FVIII deficiency to 1.9–11.3% of ...
Giang N. Nguyen, J. Everett, Samita Kafle, A. Roche, H. Raymond, Jacob S. Leiby, Christian Wood, C. Assenmacher, E. Merricks, C. T. Long, H. Kazazian, T. Nichols, F. Bushman, D. Sabatino, D. Sabatino   +14 more
semanticscholar   +1 more source

Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)

Research and Practice in Thrombosis and Haemostasis, 2022
Emicizumab is a subcutaneously administered humanized, bispecific, monoclonal antibody approved for prophylaxis in people with hemophilia A.
Renchi Yang, Shujie Wang, Xuefeng Wang, Jing Sun, A. Chuansumrit, Jianfeng Zhou, C. Schmitt, W. Hsu, Jeffrey Xu, Lindong Li, Tiffany Y. Chang, Xielan Zhao   +11 more
semanticscholar   +1 more source

Impact of novel hemophilia therapies around the world

Research and Practice in Thrombosis and Haemostasis, 2022
Hemophilia A and B are hereditary bleeding disorders, characterized by factor VIII or IX deficiencies, respectively. For many decades, prophylaxis with coagulation factor concentrates (replacement therapy) was the standard‐of‐care approach in hemophilia.
M. Ozelo, Gabriela G Yamaguti-Hayakawa
semanticscholar   +1 more source

Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia [PDF]

, 2017
The most worrying complication of replacement therapy for severe hemophilia A and B is currently the occurrence of inhibitory alloantibodies against infused factor VIII and factor IX, respectively.
Franchini, Massimo, Liumbruno, Giancarlo Maria, Marano, Giuseppe, Mengoli, Carlo, Piccinini, Vanessa, Pupella, Simonetta, Vaglio, Stefania   +6 more
core   +1 more source

The history and evolution of the clinical effectiveness of haemophilia type a treatment: a systematic review. [PDF]

, 2012
First evidence of cases of haemophilia dates from ancient Egypt, but it was when Queen Victoria from England in the 19th century transmitted this illness to her descendants, when it became known as the "royal disease".
A Aleem, A Farrugia, A Miners, A Mocroft, AL Dunn, BA Konkle, BL Evatt, BM Feldman, CA Lee, CL Poel Van der, Claudia P. Casas, D Dimichele, D Dimichele, E Berntorp, E Berntorp, E Santagostino, Hector E. Castro, J Astermark, J Goudemand, J Oldenburg, J Stachnik, J Tusell, J Wight, Juan David Rueda, K Porter, L Valentino, L Valentino, LM Aledort, M Carcao, M Carcao, M Depka Von, M Franchini, M Lloyd, M Makris, María Fernanda Briceño, MD Carcao, MV Ragni, P Petrini, PM Mannucci, PM Mannucci, R Prabhu, S Royal, SA Larsson, SC Darby, SC Gouw, T Wong, TL Chorba, VS Blanchette, WK Hoots   +48 more
core   +2 more sources

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

Journal of Thrombosis and Haemostasis, 2021
Fitusiran, an investigational small interfering RNA therapy, reduces antithrombin production to rebalance hemostasis in people with hemophilia A or B, with or without inhibitors.
K. Pasi, T. Lissitchkov, V. Mamonov, T. Mant, M. Timofeeva, C. Bagot, P. Chowdary, P. Georgiev, L. Gercheva‐Kyuchukova, Kate Madigan, H. Nguyen, Qifeng Yu, B. Mei, C. Benson, M. Ragni   +14 more
semanticscholar   +1 more source

MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA

Mediterranean Journal of Hematology and Infectious Diseases, 2020
The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino, Paul Tieu, Dr., Antony Chan   +2 more
doaj   +1 more source

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors

New England Journal of Medicine, 2018
BACKGROUND Emicizumab is a bispecific monoclonal antibody that bridges activated factor IX and factor X to replace the function of missing activated factor VIII, thereby restoring hemostasis.
J. Mahlangu, J. Oldenburg, I. Paz‐Priel, C. Négrier, M. Niggli, E. Mancuso, C. Schmitt, V. Jiménez‐Yuste, C. Kempton, C. Dhalluin, M. Callaghan, W. Buján, M. Shima, J. Adamkewicz, E. Asikanius, G. Levy, R. Kruse-Jarres   +16 more
semanticscholar   +1 more source