Results 71 to 80 of about 118,583,499 (297)

Hemophilia prevalence in Azerbaijan

, 2017
Aim. Assessment of hemophilia prevalence in Azerbaijan.Methods. Materials of the study were the data of the registry on hemophilia. Prevalence and its structure were calculated for the regions and towns of republican subordination: the number of ...
M M Kazimova, E A Kadimova
core   +1 more source

Changes over the last two decades in Hemophilia A and B management in a Tertiary Care Pediatric Centre

Clinical Epidemiology and Global Health
Objective: The treatment of Hemophilia A and B has significantly evolved. Study design: we conducted a retrospective, observational study among patients with HA and HB managed in a Pediatric Hematology and Oncology Unit for almost 22 years for a total of
M.P. Esposto, E. Bonetti, R. Balter, V. Pezzella, A. Zaccaron, V. Vitale, G. Caddeo, M. Chinello, G. Tridello, G. Poli, S. Cesaro   +10 more
doaj   +1 more source

Bringing Gene Therapy Into Real World Clinical Practice

Haemophilia, EarlyView.
ABSTRACT Introduction Adeno‐associated virus (AAV)‐based gene therapy for haemophilia has shifted therapeutic paradigms by enabling hepatic gene transfer, restoring endogenous clotting factor expression, and reducing reliance on conventional prophylactic treatments. Two products, valoctocogene roxaparvovec (haemophilia A) and etranacogene dezaparvovec (
Wolfgang Miesbach, Pratima Chowdary, Margareth C. Ozelo   +2 more
wiley   +1 more source

Animal Models of Hemophilia [PDF]

, 2012
The X-linked bleeding disorder hemophilia is caused by mutations in coagulation factor VIII (hemophilia A) or factor IX (hemophilia B). Unless prophylactic treatment is provided, patients with severe disease (less than 1% clotting activity) typically ...
Timothy C. Nichols, Roland W. Herzog, Sabatino, Denise E., Bellinger, Dwight A., Nichols, Timothy C., Paul E. Monahan, Elizabeth Merricks, Monahan, Paul E., Dwight A. Bellinger, Denise E. Sabatino, Merricks, Elizabeth, Herzog, Roland W.   +11 more
core   +1 more source

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

Haemophilia, EarlyView.
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita, Keiji Nogami, Chandrakala Shanmukhaiah   +2 more
wiley   +1 more source

Prospective collection of bleeding rate and factor usage in participants with hemophilia A and B in a noninvestigational product study

Research and Practice in Thrombosis and Haemostasis
Background: We report efficacy and select safety outcomes of current factor prophylaxis of adults with moderately severe to severe hemophilia A (HA) or hemophilia B (HB) in a large, prospective, noninvestigational product study.
Kaan Kavakli, Laurent Frenzel, Ali Bulent Antmen, Hazzaa Alzahrani, Davide Matino, Andrew D. Leavitt, Margareth C. Ozelo, Jan Astermark, Young-Shil Park, Robert Klamroth, Adam Cuker, Barbara A. Konkle, John McKay, Delphine Agathon, Li-Jung Tseng, Maria de los Angeles Resa, Jasmine Healy, Lisa J. Wilcox, Frank Plonski   +18 more
doaj   +1 more source

Identification of patients with congenital hemophilia in a large electronic health record database

, 2017
Michael Wang,1 Anissa Cyhaniuk,2 David L Cooper,3 Neeraj N Iyer3 1Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO, 2AC Analytic Solutions, Barrington, IL, 3Clinical Development, Medical and Regulatory Affairs, Novo
Wang M, Iyer NN, Cyhaniuk A, Cooper DL
core  

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Guidelines for the management of hemophilia

, 2013
Hemophilia is a rare disorder that is complex to diagnose and to manage. These evidence-based guidelines offer practical recommendations on the diagnosis and general management of hemophilia, as well as the management of complications including ...
Mahlangu, J.N., Mulder, K., Poon, M. C., Mahlangu, J. N., Kitchen, S., Ludlam, C.A., Brewer, A. K., Mauser-Bunschoten, E.P., Srivastava, A., Brewer, A.K., Poon, M.C., Key, N. S., Ludlam, C. A., Street, A., Mauser?Bunschoten, E. P., Llinas, A., Key, N.S.   +16 more
core   +1 more source

Performing Large‐Scale Genetic Analysis in the Bleeding Disorders Community

Haemophilia, EarlyView.
ABSTRACT Inherited bleeding disorders encompass a diverse group of conditions caused by genetic defects affecting coagulation factors, fibrinogen, von Willebrand factor, or platelet function. Despite major advances in quantitative and functional laboratory assays, a substantial diagnostic gap remains, particularly in patients with mild or atypical ...
Anna R. Blankstein, Sterre P. E. Willems, Saskia E. M. Schols, Rosanna Asselta, Gillian Lowe, Neil V. Morgan, Paula D. James   +6 more
wiley   +1 more source