Results 11 to 20 of about 33,363 (246)

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

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

Surgical Procedures Requiring Hospitalization and Perioperative Management for Patients with Hereditary Bleeding Disorders

Clinical Pediatric Hematology-Oncology, 2022
Background : : Hemophilia requires a lifetime care for bleeding control and complications. Although patients diagnosed with hemophilia receive factor replacement, they also experience a variety of medical problems as they age.
Hyeun Su Seo, Won Kee Ahn, Seung Min Hahn, Jung Woo Han, Chuhl Joo Lyu   +4 more
doaj   +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

Emicizumab initiation and bleeding outcomes in people with hemophilia A with and without inhibitors: A single‐center report

Research and Practice in Thrombosis and Haemostasis, 2021
Background Emicizumab, a bispecific antibody factor VIII mimetic, is approved for prophylaxis in hemophilia, and has different risks and side effects compared to factor VIII products.
Beth Boulden Warren, Adrian Chan, Marilyn Manco‐Johnson, Brian R. Branchford, Tyler W. Buckner, Genevieve Moyer, Elizabeth Gibson, Dianne Thornhill, Michael Wang, Christopher J Ng   +9 more
doaj   +1 more source

New therapies for von Willebrand disease

Blood Advances, 2019
: The management of von Willebrand disease (VWD) is based upon the dual correction of the primary hemostasis defect, due to the inherited deficiency of von Willebrand factor (VWF), and of the secondary defect of factor VIII coagulant activity (FVIII:C ...
Pier Mannuccio Mannucci
doaj   +1 more source

Prophylaxis in Hemophilia

Clinical and Biomedical Research, 2023
Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich, Dayenne Catelli, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

Turoctocog alfa pegol (N8‐GP) in severe hemophilia A: Long‐term safety and efficacy in previously treated patients of all ages in the pathfinder8 study

Research and Practice in Thrombosis and Haemostasis, 2022
Background N8‐GP (turoctocog alfa pegol; Esperoct) is a glycoPEGylated human recombinant factor VIII (FVIII). Objectives Pathfinder8 (NCT01480180) was a phase 3, multinational, open‐label, nonrandomized trial to investigate the long‐term safety and ...
Steven R. Lentz, Kaan Kavakli, Robert Klamroth, Mudi Misgav, Azusa Nagao, Alberto Tosetto, Pernille Juul Jørgensen, Marek Zak, Laszlo Nemes   +8 more
doaj   +1 more source

Hemophilia: a biography on therapeutical approaches

Clinical and Biomedical Research, 2023
The history of hemophilia is ancient, with descriptions dated to the 2nd century AD. The first modern narratives appeared in 1800s, when total blood transfusion was the only available treatment and life expectancy was remarkably low.
Dayenne Catelli, Júlia Plentz Portich, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj