Results 1 to 10 of about 24,946,900 (320)

Nonneutralizing antibodies in Nordic persons with moderate hemophilia A and B (the MoHem study) [PDF]

Research and Practice in Thrombosis and Haemostasis
Background: The impact of nonneutralizing antibodies (NNAs) in moderate hemophilia is elusive. Objectives: To explore the presence of NNAs in Nordic persons with moderate hemophilia A (MHA) and B (MHB) in relation to treatment modality, clinical outcome,
Ragnhild J. Måseide, MD, PhD, Erik Berntorp, MD, PhD, Jan Astermark, MD, PhD, Anna Olsson, MD, PhD, Maria Bruzelius, MD, PhD, Tony Frisk, MD, PhD, Vuokko Nummi, MD, PhD, Riitta Lassila, MD, PhD, Karin Strandberg, MD, PhD, Geir E. Tjønnfjord, MD, PhD, Pål A. Holme, MD, PhD   +10 more
doaj   +2 more sources

Hemophilia in pediatric age [PDF]

Romanian Journal of Pediatrics, 2023
Hemophilia represents the most common inherited bleeding disorder linked to the X chromosome, which, if not properly treated, can lead to lifelong disabilities.
Cristina Elena Singer, Carmen Simona Cosoveanu, Jaqueline Abdul-Razzak, Alina Popescu, Iulian Iliescu, Maria Madalina Singer, Mihaela Popescu, Alin Iulian-Silviu Popescu   +7 more
doaj   +1 more source

HEMATURIA IN HEMOPHILIA: WHAT DO WE KNOW? A CHALLENGING CASE STUDY AND LITERATURE REVIEW [PDF]

Euromediterranean Biomedical Journal, 2022
Hemophilia is a genetically determined bleeding disorder, which, if not properly managed, can cause lifelong disabilities. Hemorrhages in the joints and soft tissues are largely studied.
Alessandra Di Nora
doaj   +1 more source

Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.

New England Journal of Medicine, 2023
BACKGROUND Efanesoctocog alfa provides high sustained factor VIII activity by overcoming the von Willebrand factor-imposed half-life ceiling. The efficacy, safety, and pharmacokinetics of efanesoctocog alfa for prophylaxis and treatment of bleeding ...
A. von Drygalski, P. Chowdary, R. Kulkarni, Sophie Susen, B. Konkle, J. Oldenburg, D. Matino, R. Klamroth, A. Weyand, V. Jiménez‐Yuste, K. Nogami, S. Poloskey, B. Winding, A. Willemze, K. Knobe   +14 more
semanticscholar   +1 more source

Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.

New England Journal of Medicine, 2022
BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)-based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
M. Ozelo, J. Mahlangu, K. Pasi, A. Giermasz, A. Leavitt, M. Laffan, E. Symington, D. Quon, Jiaan-Der Wang, K. Peerlinck, S. Pipe, B. Madan, N. Key, G. Pierce, Brian O'Mahony, R. Kaczmarek, Joshua Henshaw, A. Lawal, K. Jayaram, Mei Huang, Xinqun Yang, W. Y. Wong, Benjamin Kim   +22 more
semanticscholar   +1 more source

In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Science Advances, 2022
Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic ...
J. Han, MinJeong Kim, B. Choi, Jeong Hyeon Lee, G. Lee, M. Jeong, Yeji Lee, Eun-Ah Kim, Hye-Kyung Oh, Nanyeong Go, Hyerim Lee, K. J. Lee, U. Kim, J. Y. Lee, Seokjoong Kim, Jun Chang, Hyukjin Lee, D. Song, S. Yeom   +18 more
semanticscholar   +1 more source

Prophylactic emicizumab for hemophilia A in the Asia‐Pacific region: A randomized study (HAVEN 5)

Research and Practice in Thrombosis and Haemostasis, 2022
Emicizumab is a subcutaneously administered humanized, bispecific, monoclonal antibody approved for prophylaxis in people with hemophilia A.
Renchi Yang, Shujie Wang, Xuefeng Wang, Jing Sun, A. Chuansumrit, Jianfeng Zhou, C. Schmitt, W. Hsu, Jeffrey Xu, Lindong Li, Tiffany Y. Chang, Xielan Zhao   +11 more
semanticscholar   +1 more source

Emicizumab-induced photosensitivity

TH Open, 2022
Emicizumab constitutes a novel and effective prophylaxis for hemophilia A patients with and without inhibitors. In this case report, we describe an emicizumab-induced photosensitivity that forced permanent sun-exposure suppression.
Pedro Asensi Cantó, Mercedes Rodríguez Serna, Pilar Lloret Madrid, Jürgen Solís Ruiz, Ana Rosa Cid Haro, Santiago Bonanad Boix, Saturnino Haya Guaita   +6 more
doaj   +1 more source

Updates on Novel Non-Replacement Drugs for Hemophilia

Pharmaceuticals, 2022
Over the last decade, the world of hemophilia has experienced an unprecedented therapeutic advance, thanks to the progress in bioengineering technologies, leading to the introduction of drugs with novel mechanisms of action based on restoring thrombin ...
Roberta Gualtierotti, Samantha Pasca, Alessandro Ciavarella, Sara Arcudi, Andrea Giachi, Isabella Garagiola, Chiara Suffritti, Simona Maria Siboni, Flora Peyvandi   +8 more
doaj   +1 more source

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

New England Journal of Medicine, 2021
BACKGROUND The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose.
Lindsey A. George, P. Monahan, M. Eyster, S. Sullivan, M. Ragni, S. Croteau, J. Rasko, M. Recht, B. Samelson-Jones, A. Macdougall, Kristen Jaworski, R. Noble, M. Curran, K. Kuranda, F. Mingozzi, Tiffany Chang, K. Reape, X. Anguela, K. High   +18 more
semanticscholar   +1 more source