Results 81 to 90 of about 12,392,515 (266)
Patient and parent preferences for characteristics of prophylactic treatment in hemophilia
Roberto Furlan,1 Sangeeta Krishnan,2 Jeffrey Vietri3 1Advanced Methods, Kantar Health, Epsom, Surrey, UK; 2Global Health Economics and Outcomes Research, Biogen, MA, USA; 3Health Outcomes, Kantar Health, Milan, Italy Introduction: New longer-acting ...
Furlan R, Krishnan S, Vietri J
doaj
Introduction: Deficiency of factor VIII (Hemophilia A), factor IX (Hemophilia B) and Von Willebrand's factor are the most frequent coagulation defects.
Sandip Kumar +5 more
doaj +1 more source
Bringing Gene Therapy Into Real World Clinical Practice
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 +2 more
wiley +1 more source
GlycoPEGylated recombinant factor IX for hemophilia B in context
Elena Santagostino, Maria Elisa Mancuso Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca’ Granda Foundation, Maggiore Hospital Policlinic, Milan, Italy Abstract: Decisions over hemophilia treatment selection and switching involve ...
Santagostino E, Mancuso ME
core
FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives
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 +2 more
wiley +1 more source
Prepared by the World Federation of Hemophilia in collaboration with the World Health Organization79 ...
World Health Organization +3 more
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Background: The large observed variability in hemophilia prevalence prevents robust estimation of burden of disease. Objective: To estimate the prevalence and prevalence at birth of hemophilia and the associated life expectancy disadvantage ...
Chambost, H. +13 more
core +1 more source
Gene Editing for Haemophilia—The Next Frontier
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 +3 more
wiley +1 more source
Spectrum of hemophilia in Diyala-Iraq
Background: Deficiencies of coagulation factors have been recognized for centuries. The most common inherited factor deficiencies are the hemophilias, X-linked diseases caused by deficiency of factor VIII (hemophilia A) or factor IX ( hemophilia B ...
Imad Ahmed Lateef +3 more
doaj +2 more sources
Clinical observations of hemophilia [PDF]
application/pdfClinical and laboratory studies were established on 7 cases with hemophilia A and 2 cases with hemophilia B. 1) The age of the patients in the first visit to our clinic were less than 5 years old, except for one case.
ヨシオカ, ケイイチロウ +20 more
core +2 more sources

