Results 21 to 30 of about 113,823 (359)
Research and Practice in Thrombosis and Haemostasis, 2021 Background Emicizumab, a bispecific antibody factor VIII mimetic, is approved for prophylaxis in hemophilia, and has different risks and side effects compared to factor VIII products.
Beth Boulden Warren +9 more
doaj +1 more source
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
Chinese Expert Consensus on Pharmacokinetics Guided Treatment for Hemophilia A
罕见病研究, 2022 Hemophilia A is an X-chromosome-linked recessive genetic disease that lacks coagulation factor Ⅷ (Factor Ⅷ, FⅧ) and is clinically manifested as spontaneous or excessive bleeding after injury.The current main treatment for hemophilia A is alternative ...
Hemophilia Treatment Center Collaborative Network of China
doaj +1 more source
Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy
Hepatology, EarlyView., 2022 Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann +25 more
wiley +1 more source
Establishment and Evolution of China National Hemophilia Registry
罕见病研究, 2022 Hemophilia is an inherited bleeding disorder and a type of rare disease that is hereditary, lifelong and disabling. The establishment of a National Hemophilia Registry is foundational to treating hemophilia.
XUE Feng, YANG Renchi
doaj +1 more source
Hematology. American Society of Hematology. Education Program, 2022 The cloning of the factor VIII (FVIII) and factor IX (FIX) genes in the 1980s has led to a succession of clinical advances starting with the advent of molecular diagnostic for hemophilia, followed by the development of recombinant clotting factor ...
A. Nathwani
semanticscholar +1 more source
Adeno-associated Virus Gene Therapy for Hemophilia.
Annual Review of Medicine, 2022 In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served as model disorders for the development of gene therapy.
Benjamin J. Samelson-Jones +1 more
semanticscholar +1 more source
Hematology, Transfusion and Cell Therapy, 2023 Objective: Factor X deficiency (FXd) is a rare coagulation disorder that can be either hereditary or acquired. Case report: We characterized patients with FXd and evaluated their bleeding patterns and treatment strategies. Methodology: This retrospective
Nigar Abdullayeva +3 more
doaj +1 more source
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.
New England Journal of Medicine, 2020 BACKGROUND Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic option for persons with hemophilia A. Efficacy and safety data include 3 years of follow-up after a single administration of AAV5-hFVIII-SQ.
K. Pasi +10 more
semanticscholar +1 more source
International recommendations on the diagnosis and treatment of acquired hemophilia A
Haematologica, 2020 Acquired hemophilia A (AHA), a rare bleeding disorder caused by neutralizing autoantibodies against coagulation factor VIII (FVIII), occurs in both men and women without a previous history of bleeding.
A. Tiede +11 more
semanticscholar +1 more source