Results 41 to 50 of about 13,301,732 (374)
Etranacogene dezaparvovec for hemophilia B gene therapy
Therapeutic Advances in Rare Disease, 2021 The treatment landscape for hemophilia has been rapidly changing with introduction of novel therapies. Gene therapy for hemophilia is a promising therapeutic option for sustained endogenous factor production to mitigate the need for prophylactic ...
C. Thornburg
semanticscholar +1 more source
In Vivo Measurement of Blood Clot Mechanics from Computational Fluid Dynamics based on Intravital Microscopy Images [PDF]
Computers in Biology and Medicine, 2019, 2018 Ischemia leading to heart attacks and strokes is the major cause of deaths in the world. Whether an occlusion occurs or not, depends on the ability of a growing thrombus to resist forces exerted on its structure. This manuscript provides the first known in vivo measurement of the stresses that clots can withstand, before yielding to the surrounding ...
arxiv +1 more source
Journal of Thrombosis and Haemostasis, 2020 An international, multicenter extension study evaluated recombinant fusion protein linking recombinant coagulation factor IX (FIX) with recombinant human albumin (rIX‐FP) in hemophilia B (FIX ≤ 2%) patients previously enrolled in a phase III study or who
M. Mancuso +7 more
semanticscholar +1 more source
Randomized trial of a DVD intervention to improve readiness to self-manage joint pain [PDF]
, 2011 Report of a randomized controlled trial of a motivational intervention to promote self-management of joint painA DVD (digital video disk) intervention to increase readiness to self-manage joint pain secondary to hemophilia was informed by a 2-phase ...
Abraham +43 more
core +2 more sources
Ultrasound Detection of Subquadricipital Recess Distension [PDF]
arXiv, 2022 Joint bleeding is a common condition for people with hemophilia and, if untreated, can result in hemophilic arthropathy. Ultrasound imaging has recently emerged as an effective tool to diagnose joint recess distension caused by joint bleeding. However, no computer-aided diagnosis tool exists to support the practitioner in the diagnosis process.
arxiv
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
Blood, 2018 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
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
Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 [PDF]
Journal of Controlled Release, 2019 Curiel, David T +5 more
core +3 more sources
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 +11 more
semanticscholar +1 more source
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 +6 more
semanticscholar +1 more source