Results 51 to 60 of about 78,600 (311)

Evolving Real‐World Data and Evidence Use for New Drugs and Regenerative Medical Products Approvals in Japan—An Analysis of the 6‐Year Trend

Clinical Pharmacology &Therapeutics, EarlyView.
Several regulatory initiatives have been made to clarify the acceptability and requirements of real‐world data and real‐world evidence (RWD/E) for the benefit/risk assessment of new medical products in Japan. The objectives of this review were to characterize the use of RWD/E in regulatory applications of new medical products and to describe the ...
Suguru Okami, Kako Shimotsumagari, Yasunari Sadatsuki   +2 more
wiley   +1 more source

Development and Validation of a Liquid Chromatography High-Resolution Mass Spectrometry Method for Blood Desmopressin Quantification and Its Application in Hemophilia A Patients. [PDF]

Rapid Commun Mass Spectrom
ABSTRACT Desmopressin (DDAVP), which indirectly increases Coagulation Factor VIII concentrations in the blood, is a common treatment for bleeding disorders such as von Willebrand disease or hemophilia A. However, DDAVP exhibits significant variability in response due to interindividual differences in pharmacokinetics.
Hodin S, Escal J, Feliu C, Pontis A, Josset L, Serban A, Guillet B, Delavenne X.   +7 more
europepmc   +2 more sources

A Comparison between Recombinant Activated Factor VII (Aryoseven) and Novoseven in Patients with Congenital Factor VII Deficiency [PDF]

, 2015
In order to establish the efficacy and biosimilar nature of AryoSeven to NovoSeven in the treatment of congenital factor VII (FVII) deficiency, patients received either agent at 30 1/4g/kg, intravenously per week for 4 weeks, in a randomized fashion. The
Abolghasemi, H., Ahmadinejad, M., Baghaeipour, M.R., Dehdezi, B.K., Eshghi, P., Faranoush, M., Fayazfar, R., Heshmat, R., Hoorfar, H., Kamyar, K., Karimi, M., Khoeiny, B., Managhchi, M., Mehrvar, A., Mirbehbahani, N.B., Naderi, M., Toogeh, G.   +16 more
core  

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]

, 2002
The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Davidoff, AM, Hanawa, H, Hoffer, FA, Hu, Y, Lozier, JN, Mandrell, TD, Nathwani, AC, Ng, CYC, Nienhuis, AW, Nikanorov, A, Slaughter, C, Vanin, EF, Zhou, J   +12 more
core   +1 more source

Evaluation of coagulation disorders and iron deficiency in women with heavy menstrual bleeding

International Journal of Gynecology &Obstetrics, EarlyView.
Abstract Objective To explore the incidence of congenital bleeding disorders (CBD), which may result in anemia, in a large cohort of women referred for heavy menstrual bleeding (HMB) in a specialized gynecologic unit. Methods Between January 2022 and January 2024, all women referred for HMB were screened.
Lucia Rugeri, Yelena Fruchou, Caroline Brodbeck, Marianne Bastien, Amandine Lioux, Giulia Gouy, Marion Cortet, Gil Dubernard, Yesim Dargaud   +8 more
wiley   +1 more source

Disclosure of Maternal HIV Status to Children: To Tell or Not To Tell . . . That Is the Question [PDF]

, 2007
HIV-infected mothers face the challenging decision of whether to disclose their serostatus to their children. From the perspective of both mother and child, we explored the process of disclosure, providing descriptive information and examining the ...
A. Shaffer, B. J. Antle, B. Rime, B. W. C. Forsyth, C. Finkenauer, C. Finkenauer, C. Nostlinger, C. S. Edelbrock, D. A. Murphy, D. A. Murphy, D. A. Murphy, D. A. Murphy, D. A. Murphy, D. J. Pilowsky, D. Melvin, E. W. Schrimshaw, G. E. Wyatt, J. Faithfull, J. M. Simoni, J. Ware, K. E. Grant, K. J. Petrie, L. Armistead, L. Armistead, L. Armistead, L. Armistead, L. J. Bauman, L. Moneyham, L. S. Radloff, L. S. Wiener, M. J. Rotheram-Borus, M. L. Lee, M. Lipson, P. Lester, R. A. Ostrom, R. L. Sowell, R. N. Jamison, S. Dorsey, S. Esposito, S. F. Nagler, S. Harter, T. L. Tompkins, T. M. Achenbach, T. M. Achenbach, T. M. Achenbach, Tanya L. Tompkins, V. N. Niebuhr   +46 more
core   +3 more sources

FIGO good practice recommendations: Assisted vaginal birth and the second stage of labor

International Journal of Gynecology &Obstetrics, EarlyView.
Abstract Cesarean birth (CB) rates are increasing globally, with a current prevalence of 21.1% and a projected prevalence of 28.5% by 2030. Conversely, assisted vaginal birth (AVB) rates are declining, with reported rates of less than 10% in low‐ and middle‐income countries (LMICs) and 10%–15% in high‐income countries (HICs). In some African countries,
Akaninyene E. Ubom, Eytan R. Barnea, Nicoletta DiSimone, Martin Mueller, Jolly Beyeza‐Kashesya, Ines Nunes, Albaro J. Nieto‐Calvache, Elif G. Topcu, Zechariah J. Malel, Bo Jacobsson, Alison Wright, the FIGO Childbirth and Postpartum Hemorrhage Committee   +11 more
wiley   +1 more source

Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A. [PDF]

, 2012
nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Abriss, Daniela, Milanov, Peter, Pepperkok, Rainer, Quade-Lyssy, Patricia, Roth, Stefanie D., Schüttrumpf, Jörg, Seifried, Erhard, Simpson, Jeremy C., Tonn, Torsten, Ungerer, Christopher   +9 more
core   +2 more sources

Acquired Haemophilia Occurring in Association With Bullous Pemphigoid

JEADV Clinical Practice, EarlyView.
ABSTRACT Acquired haemophilia A (AHA) is a rare condition due to neutralizing antibodies against Factor VIII and can result in severe bleeding manifestations. The association of AHA with bullous pemphigoid (BP) is rare, most frequently observed in the elderly, and has been attributed to cross‐reactive antibodies targeting Factor VIII and BPAG2 protein.
Wen Yang Benjamin Ho, Zi Teng Chai, Haur Yueh Lee, Yen Loo Lim   +3 more
wiley   +1 more source

The association between unemployment and treatment among adults with hemophilia

Research and Practice in Thrombosis and Haemostasis
Background: People with hemophilia often experience pain and suffer from comorbidities related to their bleeding disorder. Consequently, unemployment due to disability is prevalent among people with hemophilia. Objectives: To explore associations between
Christian Qvigstad, Lars Q. Sørensen, Geir E. Tjønnfjord, Pål André Holme, Ingrid Pabinger, Cedric Hermans, Roseline d’Oiron, Robert Klamroth, Johannes Oldenburg, Natascha Marquardt, Peter Staritz, Olga Katsarou, Uri Martinowitz, Aharon Lubetsky, Gili Kenet, Annarita Tagliaferri, Maria Elisa Mancuso, Roger Schutgens, Pål André HolmE, Jerzy Windyga, Irena Zupan, Victor Jimenez Yuste, Ramiro Nunez, Philippe de Moerloose, Erik Berntorp, Jan Astermark, Campbell Tait, Gerry Dolan   +27 more
doaj   +1 more source