Results 11 to 20 of about 13,000,271 (286)
BMC Health Services Research, 2022 Background Hemophilia care in mainland China has been greatly improved since the establishment of the Hemophilia Treatment Center Collaborative Network of China (HTCCNC), and most of drugs for hemophilia have been covered by basic medical insurance ...
Zhengwei Huang +6 more
doaj +1 more source
Phase 1-2 Trial of AAVS3 Gene Therapy in Patients with Hemophilia B.
New England Journal of Medicine, 2022 BACKGROUND FLT180a (verbrinacogene setparvovec) is a liver-directed adeno-associated virus (AAV) gene therapy that uses a synthetic capsid and a gain-of-function protein to normalize factor IX levels in patients with hemophilia B.
P. Chowdary +17 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
Current Medical Research and Opinion, 2022 Objective Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life.
Jinesh Shah +4 more
semanticscholar +1 more source
The Molecular Basis of FIX Deficiency in Hemophilia B
International Journal of Molecular Sciences, 2022 Coagulation factor IX (FIX) is a vitamin K dependent protein and its deficiency causes hemophilia B, an X-linked recessive bleeding disorder. More than 1000 mutations in the F9 gene have been identified in hemophilia B patients.
G. Shen, Meng Gao, Qing Cao, Weikai Li
semanticscholar +1 more source
Seroprevalence to adeno‐associated virus type 6 in people with hemophilia B from a UK adult cohort
Research and Practice in Thrombosis and Haemostasis, 2022 Background Gene therapy shows promise as a potential “cure” for hemophilia A and B. Adeno‐associated virus (AAV) vectors are the leading platform to deliver modified genetic code of factor VIII or IX to the liver effecting endogenous production.
Sara Boyce +9 more
doaj +1 more source
A Case of Acquired Hemophilia A and Congenital Hemophilia B
Cureus, 2022 Congenital hemophilia B is a rare, inherited X-linked bleeding disorder caused by a deficiency of factor IX (FIX). Acquired hemophilia A is a rare, acquired bleeding disorder which presents as new onset bleeding in older adults due to the development of autoantibodies against factor VIII (FVIII).
Fortier, Julia C +4 more
openaire +2 more sources
Nature Communications, 2022 AAV-delivered CRISPR/Cas9 (AAV-CRISPR) has shown promising potentials in preclinical models to efficiently insert therapeutic gene sequences in somatic tissues.
Xiangjun He +16 more
semanticscholar +1 more source
Hämostaseologie, 2022 Hemophilia A and hemophilia B are rare congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively.
B. Pezeshkpoor, J. Oldenburg, A. Pavlova
semanticscholar +1 more source
Blood Advances, 2021 PUPs B-LONG evaluated the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients (PUPs) with hemophilia B. In this open-label, phase 3 study, male PUPs (age
B. Nolan +12 more
semanticscholar +1 more source