Results 51 to 60 of about 49,324 (252)

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

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  

Anti-CD20 as the B-Cell Targeting Agent in a Combined Therapy to Modulate Anti-Factor VIII Immune Responses in Hemophilia a Inhibitor Mice [PDF]

, 2014
Neutralizing antibody formation against transgene products can represent a major complication following gene therapy with treatment of genetic diseases, such as hemophilia A.
Carol H. Miao, Chao Lien Liu, Chérie L. Butts, Jacqueline Lin, Peiqing Ye   +4 more
core   +1 more source

Cost‐effectiveness of fenfluramine as add‐on treatment in the management of Dravet Syndrome: A real‐world multicenter study

Epilepsia Open, EarlyView.
Abstract Objective Dravet syndrome (DS) is a rare disease with a high clinical and socioeconomic impact on patients, society, and the healthcare system. The recent approval of therapies such as fenfluramine (FFA) has transformed the treatment landscape; however, data on their cost‐effectiveness are still scarce. This study evaluates the real‐world cost‐
Paolo A. Cortesi, Carla Fornari, Alessandra Boncristiano, Rita Facchetti, Simona Balestrini, Viola Doccini, Nicola Specchio, Nicola Pietrafusa, Marina Trivisano, Francesca Darra, Alberto Cossu, Domenica I. Battaglia, Michela Quintiliani, Maria L. Gambardella, Lorenzo G. Mantovani, Renzo Guerrini   +15 more
wiley   +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

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

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

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