Results 31 to 40 of about 14,161,714 (418)
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate.
Wolfgang A Miesbach+13 more
semanticscholar +1 more source
Triangulated Loop Quantum Cosmology: Bianchi IX and inhomogenous perturbations [PDF]
We develop the "triangulated" version of loop quantum cosmology, recently introduced in the literature. We focus on the "dipole" cosmology, where space is a three-sphere and the triangulation is formed by two tetrahedra. We show that the discrete fiducial connection has a simple and appealing geometrical interpretation and we correct the ansatz on the ...
arxiv +1 more source
Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia [PDF]
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+6 more
core +1 more source
A global phase 3 study evaluated the pharmacokinetics, efficacy, and safety of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in 63 previously treated male patients (12-61 years) with severe hemophilia B (factor IX [FIX ...
E. Santagostino+16 more
semanticscholar +1 more source
The X‐linked genetic bleeding disorder caused by deficiency of coagulator factor IX, hemophilia B, is a disease ideally suited for gene therapy with genome editing technology. Here, we identify a family with hemophilia B carrying a novel mutation, Y371D,
Yuting Guan+17 more
semanticscholar +1 more source
Seroprevalence of Hepatitis B and Hepatitis C in People with Haemophilia at a Tertiary Care Centre, Assam, India: A Cross-sectional Study [PDF]
Introduction: Prior to the advent of recombinant factor products, the treatment of haemophilia was mainly based on the administration of direct blood products like whole blood, Fresh Frozen Plasma (FFP), and cryoprecipitates. This led to a high incidence
Anupam Dutta+2 more
doaj +1 more source
Extended Half-Life Factor VIII and Factor IX Preparations
In the last couple of years, several extended half-life factor VIII and factor IX preparations were intensively studied and gained approval. In order to extend half-lives, techniques like fusion to protein conjugates (Fc part of IgG1 or albumin ...
L. Graf
semanticscholar +1 more source
Experimental Mg IX photorecombination rate coefficient [PDF]
The rate coefficient for radiative and dielectronic recombination of berylliumlike magnesium ions was measured with high resolution at the Heidelberg heavy-ion storage ring TSR. In the electron-ion collision energy range 0-207 eV resonances due to 2s -> 2p (Delta N = 0) and 2s -> 3l (Delta N=1) core excitations were detected. At low energies below 0.15
arxiv +1 more source
Molecular genetic study of hemophilia B in an Algerian population [PDF]
Hemophilia B is inherited as x-linked recessive disorder, carried by females, where males are affected.Rare cases of females affected with hemophilia B are known.
Abla, Zidani+4 more
core +2 more sources
Factor Activity Assays for Monitoring Extended Half-Life FVIII and Factor IX Replacement Therapies
The advent of modified factor VIII (FVIII) and factor IX (FIX) molecules with extended half-lives (EHLs) compared with native FVIII and FIX represents a major advance in the field of hemophilia care, with the potential to reduce the frequency of ...
S. Kitchen, S. Tiefenbacher, R. Gosselin
semanticscholar +1 more source