Results 31 to 40 of about 13,284,040 (264)

Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B.

Blood Advances, 2021
PUPs B-LONG evaluated the safety and efficacy of recombinant factor IX Fc fusion protein (rFIXFc) in previously untreated patients (PUPs) with hemophilia B. In this open-label, phase 3 study, male PUPs (age
B. Nolan, A. Klukowska, A. Shapiro, A. Rauch, M. Recht, M. Ragni, J. Curtin, S. Gunawardena, Sutirtha Mukhopadhyay, D. Jayawardene, B. Winding, K. Fischer, R. Liesner   +12 more
semanticscholar   +1 more source

The frequency of joint hemorrhages and procedures in nonsevere hemophilia A vs B

Blood Advances, 2018
: Data are needed on minimal factor activity (FA) levels required to prevent bleeding in hemophilia. We aimed to evaluate associations between hemophilia type and FA level and joint bleeding and orthopedic procedures using longitudinal data.
J. Michael Soucie, Paul E. Monahan, Roshni Kulkarni, Barbara A. Konkle, Marshall A. Mazepa   +4 more
doaj   +1 more source

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.

Molecular Therapy, 2020
Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene transfer highlights the need to answer outstanding clinical questions of safety, durability ...
Lindsey A. George, M. Ragni, J. Rasko, L. Raffini, Benjamin J. Samelson-Jones, Margareth C. Ozelo, Maria Hazbon, Alexa R. Runowski, J. Wellman, Katie Wachtel, Yifeng Chen, X. Anguela, K. Kuranda, F. Mingozzi, K. High   +14 more
semanticscholar   +1 more source

The Cost-effectiveness of Gene Therapy for Severe Hemophilia B: Microsimulation Study from the United States Perspective.

Blood, 2021
Adeno-associated virus (AAV)-mediated gene therapy is a novel treatment promising to reduce morbidity associated with hemophilia. While multiple clinical trials continue to evaluate efficacy and safety, limited cost-effectiveness data have been published.
Nancy S. Bolous, Yichen Chen, Huiqi Wang, A. Davidoff, M. Devidas, Timothy W Jacobs, M. Meagher, A. Nathwani, E. Neufeld, Bryan A. Piras, C. Rodríguez-Galindo, U. Reiss, N. Bhakta   +12 more
semanticscholar   +1 more source

MOLECULAR MECHANISMS OF INHIBITOR DEVELOPMENT IN HEMOPHILIA

Mediterranean Journal of Hematology and Infectious Diseases, 2020
The development of neutralizing antibodies in hemophilia is a serious complication of factor replacement therapy. These antibodies, also known as “inhibitors”, significantly increase morbidity within the hemophilia population and lower the quality of ...
Davide Matino, Paul Tieu, Dr., Antony Chan   +2 more
doaj   +1 more source

Inhibitor incidence in an unselected cohort of previously untreated patients with severe hemophilia B: a PedNet study

Haematologica, 2020
The incidence of factor IX (FIX) inhibitors in severe hemophilia B (SHB) is not well defined. Frequencies of 3-5% have been reported but most studies to date have been small, including patients with different severities, and without prospective follow up
C. Male, N. Andersson, A. Rafowicz, R. Liesner, K. Kurnik, K. Fischer, H. Platokouki, E. Santagostino, H. Chambost, B. Nolan, C. Königs, G. Kenet, R. Ljung, M. H. Van den Berg   +13 more
semanticscholar   +1 more source

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

New England Journal of Medicine, 2014
BACKGROUND In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months.
A. Nathwani, U. Reiss, E. Tuddenham, Cecilia Rosales, P. Chowdary, J. McIntosh, M. della Peruta, Elsa Lhériteau, Nishali Patel, D. Raj, A. Riddell, J. Pie, S. Rangarajan, D. Bevan, M. Recht, Yu-min P Shen, K. Halka, E. Basner-Tschakarjan, F. Mingozzi, K. High, J. Allay, M. Kay, C. Ng, Junfang Zhou, M. Cancio, C. Morton, J. Gray, D. Srivastava, A. Nienhuis, A. Davidoff   +29 more
semanticscholar   +1 more source

A Case of Hemophilia B with Abdominal and Pelvic Pseudotumor

罕见病研究, 2022
Hemophilia B is a genetic disorder caused by coagulation factor Ⅸ(FⅨ) deficiency, mainly manifesting as joint, muscle and deep tissue bleeding. Hemophilia pseudotumor is a mass formed by soft tissue liquefaction and necrosis caused by repeated bleeding ...
ZHANG Jing, JU Mankai, ZHANG Kemin, CHEN Lingling, WANG Yuhua, ZHANG Lei, YANG Renchi, XUE Feng   +7 more
doaj   +1 more source

Health care resource utilization and cost burden of hemophilia B in the United States.

Blood Advances, 2021
Hemophilia B is a rare congenital blood disorder characterized by factor IX deficiency. Clinical profiles of hemophilia B range from mild to severe forms of the disease.
T. Buckner, I. Bocharova, Kaitlin A. Hagan, A. Bensimon, Hongbo Yang, E. Wu, E. Sawyer, Nanxin Li   +7 more
semanticscholar   +1 more source

Cochlear implantation in hemophilia B—a rare case report

The Egyptian Journal of Otolaryngology, 2022
Background Hemophilia B is an X-linked inherited disease, mainly caused by deficiency of factor IX. Severity of the disease is manifested by the factor IX deficiency in the blood.
Chetan Yadav, Diva Sharma, Neeraj Suri, Meenesh Juvekar   +3 more
doaj   +1 more source