Results 41 to 50 of about 69,645 (303)
Journal of Thrombosis and Haemostasis, 2010 Mild hemophilia A (HA), defined by clinical features and factor VIII coagulant activity (FVIII:C) between 0.05 and 0.40 IU mL(-1), is characteristically distinct from severe HA. Indeed, although the molecular characterization of mild HA has permitted the identification of specific underlying mutations, its clinical phenotype is strikingly different ...
Franchini M +2 more
openaire +3 more sources
Hemophilia Gene Therapy: Approaching the First Licensed Product
HemaSphere, 2021 The clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product.
P. Batty, D. Lillicrap
semanticscholar +1 more source
Romanian Journal of Laboratory Medicine, 2020 The development of factor VIII inhibitors (allo-antibodies) continues to be a major complication in the management of severe forms of hemophilia A, especially as far as treatment and treatment response monitoring is concerned.
Melen Brinza +6 more
doaj +1 more source
Gene Therapy in Hemophilia: Recent Advances
International Journal of Molecular Sciences, 2021 Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors.
E. Rodríguez‐Merchán +2 more
semanticscholar +1 more source
Oman Journal of Ophthalmology, 2009 Hemophilic pseudotumor is an uncommon complication of factor VIII and IX deficiencies in the coagulation cascade and occurs in a wide spectrum of bones and soft tissues. We present a six-year-old boy with hemophilic pseudotumor localized in the right orbit.
Pooja Sethi +3 more
openaire +4 more sources
Hemophilia therapy: the future has begun
Haematologica, 2020 The success story of hemophilia care first began in the 1970s, when the availability of plasma-derived concentrates of coagulation factor VIII (FVIII) and factor IX (FIX) provided efficacious treatment of bleeding in patients with hemophilia A and B ...
P. Mannucci
semanticscholar +1 more source
Recent Advances in the Treatment of Hemophilia: A Review
Biologics : targets & therapy, 2021 Progress in hemophilia therapy has been remarkable in the first 20 years of the third millennium, but the innovation began with the description the fractionation of plasma in 1946.
E. Marchesini, M. Morfini, L. Valentino
semanticscholar +1 more source
Blood, 2021 Intracranial hemorrhage (ICH) is a severe complication that is relatively common among hemophilia patients. This systematic review aimed to obtain more precise estimates of ICH incidence and mortality in hemophilia, which may be important for patients ...
A. Zwagemaker +9 more
semanticscholar +1 more source
Managing Relevant Clinical Conditions of Hemophilia A/B Patients
Hematology Reports, 2023 The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini +9 more
doaj +1 more source