Results 51 to 60 of about 13,000,271 (286)

Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial.

Blood, 2016
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, U. Martinowitz, T. Lissitchkov, Brigitte Pan-Petesch, H. Hanabusa, J. Oldenburg, L. Boggio, C. Négrier, I. Pabinger, Mario von Depka Prondzinski, C. Altisent, G. Castaman, Koji Yamamoto, M. Álvarez‐Román, Christine Voigt, N. Blackman, Iris Jacobs   +16 more
semanticscholar   +1 more source

Techno‐Economic Analysis of Membrane‐Based Purification Platforms for AAV Vector Production

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Technologies for large‐scale manufacturing of viral vectors for gene therapies, such as tangential flow filtration and membrane chromatography, are under development. In these early stages of process development, techno‐economic analyses are useful for identifying membrane properties yielding the greatest impact on process performance. In this
Juan J. Romero, Eleanor W. Jenkins, Jacob I. Monroe, S. Ranil Wickramasinghe, Xianghong Qian, Dibakar Bhattacharyya, Scott M. Husson   +6 more
wiley   +1 more source

Successful correction of hemophilia by CRISPR/Cas9 genome editing in vivo: delivery vector and immune responses are the key to success

EMBO Molecular Medicine, 2016
Hemophilia B is a serious hemostasis disorder due to mutations of the factor IX gene in the X chromosome. Gene therapy has gained momentum in recent years as a therapeutic option for hemophilia B.
Tuan Huy Nguyen, Ignacio Anegon
doaj   +1 more source

A study to determine the prevalence, clinical profile and incidence of formation of inhibitors in patients of hemophilia in north eastern part of India

Journal of Family Medicine and Primary Care, 2019
Introduction: Deficiency of factor VIII (Hemophilia A), factor IX (Hemophilia B) and Von Willebrand's factor are the most frequent coagulation defects.
Sandip Kumar, Sachidanand Sinha, Anju Bharti, Lalit P Meena, Vineeta Gupta, Jyoti Shukla   +5 more
doaj   +1 more source

Blood clot inspired pore‐gradient nanofiber sponge with “outer filtration” and “inner adsorption” bifunction for rapid stop of uncontrolled hemorrhage

BMEMat, EarlyView.
In this study, by mimicking the nanofiber structure and netting blood cells function of fibrin network in blood clots, we developed a novel bioinspired quaternized chitosan nanofiber sponge with distinct blood cell filtration and blood plasma absorption bifunction for rapid hemostasis.
Fujin Zhou, Yong Yang, Yixuan Li, Ruolin Cao, Zheng Chen, Zhi Wang, Naiwen Tan, Lei Tian, Botao Song   +8 more
wiley   +1 more source

Increased Thrombogenicity is Associated With Coronary Microvascular Dysfunction in Patients With STEMI—A Proof‐of‐Concept Study

Catheterization and Cardiovascular Interventions, EarlyView.
ABSTRACT Background Up to 50% of patients with ST‐segment elevation myocardial infarction (STEMI) undergoing primary percutaneous coronary intervention (pPCI) develop coronary microvascular dysfunction (CMD). Aims This study aims to assess whether a prothrombotic state of coronary blood, defined by thromboelastography (TEG), is associated with post ...
Roberto Scarsini, Denis Leonardi, Andrea Bottardi, Concetta Mammone, Leonardo Portolan, Caterina Butturini, Verdiana Galli, Francesco Della Mora, Alessandro Ruzzarin, Sara Pazzi, Anna Piccoli, Simone Fezzi, Domenico Tavella, Gabriele Pesarini, Leonardo Gottin, Flavio Ribichini   +15 more
wiley   +1 more source

Strategies for Hemophilia Treatment, a literature review of current evidence

مجله دانشگاه علوم پزشکی بیرجند, 2023
Hemophilia is an inherited bleeding disorder caused by malfunctioning or lacking blood coagulation factor VIII (hemophilia A) or IX (hemophilia B). Currently, the main treatments for these X-linked diseases are replacement therapy using periodic and ...
Fahimeh Ghasemi, Nafiseh Erfanian, Samira Karbasi, Alireza Zomorodipour   +3 more
doaj  

POLYMORPHISM OF THROMBOPHILIA GENES AND THEIR ROLE IN DEVELOPMENT OF DIFFERENT DISEASE PHENOTYPES AND THROMBOTIC COMPLICATIONS IN HEMOPHILIA PATIENTS

Сибирский научный медицинский журнал, 2019
Objective was to study genetic markers of thrombophilia in patients with hemophilia, which can affect the course of the disease and contribute to thrombotic complications. Material and methods.
T. Yu. Polyanskaya, I. S. Fevraleva, E. E. Karpov, N. V. Sadykova, M. S. Sampiev, A. V. Golobokov, G. V. Mishin, D. Yu. Petrovskiy, A. A. Koroleva, G. M. Galstyan, A. B. Sudarikov, V. Yu. Zorenko   +11 more
doaj   +1 more source

The frequency of complications in a cohort of patients diagnosed with hemophilia A and hemophilia B receiving prophylactic treatment in Colombia: A retrospective noninterventional study.

PLoS ONE, 2023
IntroductionHemophilia A and B are disorders associated with the deficit of coagulation factors VIII and IX.ObjectiveWas to determine the incidence of complications in a cohort of patients diagnosed with moderate and severe hemophilia A or B under ...
Jorge E Machado Alba, Juan David Wilches-Gutierrez, Diana Rocio Arias-Osorio, Juan Manuel Reyes, Maria Lourdes Nakandakari, Harrison David Ospina-Arzuaga, Andres Gaviria-Mendoza, Natalia Castaño-Gamboa, Luis Fernando Valladales-Restrepo, Manuel E Machado-Duque   +9 more
doaj   +1 more source

Model‐Informed Drug Development Applications and Opportunities in mRNA‐LNP Therapeutics

Clinical Pharmacology &Therapeutics, EarlyView.
The utilization of lipid nanoparticles (LNP) for encapsulating mRNA has revolutionized the field of therapeutics, enabling the rapid development of COVID‐19 vaccines and cancer vaccines. However, the clinical development of mRNA‐LNP therapeutics faces numerous challenges due to their complex mechanisms of action and limited clinical experience.
Jiawei Zhou, Rohit Rao, Monica E. Shapiro, Nessy Tania, Cody Herron, Cynthia J. Musante, Jim H. Hughes   +6 more
wiley   +1 more source