Results 71 to 80 of about 13,301,732 (374)

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

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

Perinatal Gene Transfer to the Liver [PDF]

, 2011
The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM, Chan, JK, Howe, SJ, McKay, TR, Rahim, AA, Waddington, SN, Ward, NJ   +6 more
core   +1 more source

Prenatal and molecular diagnosis of hemophilia B [PDF]

American Journal of Hematology, 1996
Prenatal diagnosis was carried out on a woman who had previously given birth to a son with a spontaneous mutation of C-->T transition at nt 31133 of the factor IX (F.IX) gene. The diagnosis was performed on chorionic villi sampling by the method of amplification-created restriction site (ACRS).
Jyh-Pyng Gau, Ji-Hsiung Young, Han-Tien Hu, Jyh-Chwan Wang   +3 more
openaire   +3 more sources

Impact of Model‐Informed Drug Development on Drug Development Cycle Times and Clinical Trial Cost

Clinical Pharmacology &Therapeutics, EarlyView.
Model‐informed drug development (MIDD) integrates data to quantify benefit/risk informing objective drug discovery and development decisions. An additional critical benefit of MIDD is postulated to be improvement in trial and program efficiencies. While the application of MIDD has grown, there have been no clear examples across programs to demonstrate ...
Vaishali Sahasrabudhe, Timothy Nicholas, Gianluca Nucci, Cynthia J Musante, Brian Corrigan   +4 more
wiley   +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

Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]

, 2001
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A, Hanawa, H, Nathwani, AC, Nienhuis, AW, Vanin, EF, Zhou, JF   +5 more
core   +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

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