Results 21 to 30 of about 13,301,732 (374)

Managing Relevant Clinical Conditions of Hemophilia A/B Patients

Hematology Reports, 2023
The Medical Directors of nine Italian Hemophilia Centers reviewed and discussed the key issues concerning the replacement therapy of hemophilia patients during a one-day consensus conference held in Rome one year ago. Particular attention was paid to the
Massimo Morfini, Jacopo Agnelli Giacchiello, Erminia Baldacci, Christian Carulli, Giancarlo Castaman, Anna Chiara Giuffrida, Giuseppe Malcangi, Angiola Rocino, Sergio Siragusa, Ezio Zanon   +9 more
doaj   +1 more source

Comprehensive analysis and prediction of long-term durability of factor IX activity following etranacogene dezaparvovec gene therapy in the treatment of hemophilia B

Current Medical Research and Opinion, 2022
Objective Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life.
Jinesh Shah, Hongseok Kim, K. Sivamurthy, P. Monahan, Michael Fries   +4 more
semanticscholar   +1 more source

The Molecular Basis of FIX Deficiency in Hemophilia B

International Journal of Molecular Sciences, 2022
Coagulation factor IX (FIX) is a vitamin K dependent protein and its deficiency causes hemophilia B, an X-linked recessive bleeding disorder. More than 1000 mutations in the F9 gene have been identified in hemophilia B patients.
G. Shen, Meng Gao, Qing Cao, Weikai Li
semanticscholar   +1 more source

Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response

Nature Communications, 2022
AAV-delivered CRISPR/Cas9 (AAV-CRISPR) has shown promising potentials in preclinical models to efficiently insert therapeutic gene sequences in somatic tissues.
Xiangjun He, Zhenjie Zhang, Jun Xue, Yaofeng Wang, Siqi Zhang, Junkang Wei, Chenzi Zhang, Jue Wang, Brian Anugerah Urip, Chun Christopher Ngan, Junjiang Sun, Yuefeng Li, Zhiqian Lu, Hui Zhao, D. Pei, Chi-kong Li, Bo Feng   +16 more
semanticscholar   +1 more source

Seroprevalence to adeno‐associated virus type 6 in people with hemophilia B from a UK adult cohort

Research and Practice in Thrombosis and Haemostasis, 2022
Background Gene therapy shows promise as a potential “cure” for hemophilia A and B. Adeno‐associated virus (AAV) vectors are the leading platform to deliver modified genetic code of factor VIII or IX to the liver effecting endogenous production.
Sara Boyce, Izabela James, Savita Rangarajan, Nicola Curry, Catherine Bagot, Steven Austin, Mike Laffan, Sarah Mangles, Kandiah Chandrakumaran, Carina Mundy   +9 more
doaj   +1 more source

Insights into the Molecular Genetic of Hemophilia A and Hemophilia B: The Relevance of Genetic Testing in Routine Clinical Practice

Hämostaseologie, 2022
Hemophilia A and hemophilia B are rare congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively.
B. Pezeshkpoor, J. Oldenburg, A. Pavlova
semanticscholar   +1 more source

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy

Hepatology, EarlyView., 2022
Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann, Marti Cabanes‐Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien‐Huber, Ahmed Abdelrahman, Xuan‐Khang Vu, Liang Zhang, Ann‐Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez‐Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwäble, Leszek Lisowski, Hildegard Büning   +25 more
wiley   +1 more source

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 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.
N. 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

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, K. Hagan, A. Bensimon, Hongbo Yang, E. Wu, E. Sawyer, Nanxin Li   +7 more
semanticscholar   +1 more source