Results 51 to 60 of about 13,086,034 (342)

Von Willebrand Disease [PDF]

, 2017
Von Willebrand Disease (VWD) is a blood clotting disorder characterized by low levels of the Von Willebrand Factor (VWF) in the blood. VWF is functions to bind platelets and promote clotting of the blood when vascular injury occurs.
Misla David, Ramon
core   +2 more sources

Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.

Blood Advances, 2019
Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific promoter.
Annette von Drygalski, A. Giermasz, G. Castaman, N. Key, S. Lattimore, F. Leebeek, Wolfgang A. Miesbach, M. Recht, A. Long, R. Gut, E. Sawyer, S. Pipe   +11 more
semanticscholar   +1 more source

Nonacog beta pegol (N9‐GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients

Research and Practice in Thrombosis and Haemostasis, 2020
We report the first analysis of an extended half‐life recombinant factor IX, nonacog beta pegol (N9‐GP), in previously untreated patients (PUPs) and minimally treated patients with hemophilia B.
Anthony K. C. Chan, J. Alamelu, C. Barnes, A. Chuansumrit, M. Garly, R. M. Meldgaard, G. Young   +6 more
semanticscholar   +1 more source

Preclinical evaluation of a next-generation, subcutaneously administered, coagulation factor IX variant, dalcinonacog alfa.

PLoS ONE, 2020
IntroductionThe rapid clearance of factor IX necessitates frequent intravenous administrations to achieve effective prophylaxis for patients with hemophilia B.
Timothy C Nichols, Howard Levy, Elizabeth P Merricks, Robin A Raymer, Martin L Lee   +4 more
doaj   +1 more source

Immune tolerance induction in a severe hemophilia B child with low titer inhibitors

Journal of Applied Hematology, 2022
Hemophilia B is an X-linked inherited bleeding disorder caused by either the absence or reduced biosynthesis of clotting factor IX (FIX). This disorder affects approximately 1 in 30,000 male individuals worldwide. Patients with severe form (FIX
Asia Almulla, Najam Awan, Faisl Khanani
doaj   +1 more source

Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B.

Human Gene Therapy, 2020
Although recombinant adeno-associated virus serotype 8 (AAV8) and serotype 5 (AAV5) vectors have shown efficacy in Phase 1 clinical trials for gene therapy of hemophilia B, it has become increasingly clear that these serotypes are not optimal for ...
Harrison C. Brown, C. Doering, R. Herzog, Chen Ling, D. Markusic, H. Spencer, Alok Srivastava, Arun Srivastava   +7 more
semanticscholar   +1 more source

Laparoscopic nephrectomy in a hemophilia B patient [PDF]

Central European Journal of Urology, 2016
Surgery in patients with hemophilia is a serious challenge. It requires a comprehensive approach, as well as careful postoperative monitoring. We present here the first case of a transperitoneal laparoscopic radical nephrectomy (TLRN) for renal cell carcinoma, of the clear-cell type, performed in a hemophilia B patient.
Gajda, Slawomir, Szopiński, Tomasz, Szczepanik, Andrzej B., Sosnowski, Roman, Szczepanik, Anna M.   +4 more
openaire   +4 more sources

Cardiac surgery in patients with Hemophilia:is it safe?

Journal of Cardiothoracic Surgery, 2020
Background The life expectancy of hemophiliacs is similar to that of the general population. As a result, the prevalence of age-related cardiovascular diseases has increased.
Amjad Shalabi, Erez Kachel, Alexander Kogan, Leonid Sternik, Liza Grosman-Rimon, Ronny Ben-Avi, Diab Ghanem, Eyalon Ram, Ehud Raanani, Mudi Misgav   +9 more
doaj   +1 more source

A natural choice for hemophilia B [PDF]

Blood, 2015
In this issue of Blood, Crudele et al describe a novel study of adeno-associated virus (AAV) vector-mediated gene therapy that induced immune tolerance to factor IX (FIX) in a hemophilia B (HB) dog with previously formed anti-FIX inhibitor antibodies (IAs).
openaire   +3 more sources

A Case of Heel Reconstruction with a Reverse Sural Artery Flap in a Hemophilia B Patient [PDF]

Archives of Plastic Surgery, 2012
Hemophilia B is a rare blood coagulation disorder. Complications such as bleeding and hematoma can cause necrosis of flaps, wound disruption, and the disturbance of wound healing.
Byung Kwon Lee, Jeong Su Shim
doaj   +2 more sources