Results 11 to 20 of about 48,361 (234)

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

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  

Practices and challenges for hemophilia management under resource constraints in Thailand

Orphanet Journal of Rare Diseases, 2023
Hemophilia is an inherited bleeding disorder caused by deficiency of a specific coagulation factor. Factor VIII deficiency is responsible for hemophilia A while factor IX deficiency is responsible for hemophilia B. As per the 2020 annual global survey by
Chatphatai Moonla, Darintr Sosothikul, Bunchoo Pongtanakul, Bundarika Suwanawiboon, Chanchai Traivaree, Rungrote Natesirinilkul, Nongnuch Sirachainan, Pantep Angchaisuksiri   +7 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  

Comorbidities, Health-Related Quality of Life, Health-care Utilization in Older Persons with Hemophilia—Hematology Utilization Group Study Part VII (HUGS VII)

Journal of Blood Medicine, 2022
Randall Curtis,1 Marilyn Manco-Johnson,2 Barbara A Konkle,3 Roshni Kulkarni,4 Joanne Wu,5 Judith R Baker,6 Megan Ullman,7 Duc Quang Tran Jr,8 Michael B Nichol5 1Factor VIII Computing, Berkeley, CA, USA; 2Hemophilia and Thrombosis Center, University of ...
Curtis R, Manco-Johnson M, Konkle BA, Kulkarni R, Wu J, Baker JR, Ullman M, Tran DQ Jr, Nichol MB   +8 more
doaj  

Interfacility Collaboration for Hemophilia Care in Japan: A Retrospective Database Study Using a Japanese Healthcare Claims Database. [PDF]

Health Sci Rep
ABSTRACT Background and Aims In Japan, about 6000 patients with hemophilia A (PwHA) are separately cared for at ~1000 clinics or hospitals. Interfacility collaboration (IFC) between hemophilia treatment centers and “satellite” medical facilities located closer to patients is necessary to eliminate care disparities.
Kinai E, Yamaguchi M, Shirahata A.
europepmc   +2 more sources

Hemophilia management: Huge impact of a tiny difference

Research and Practice in Thrombosis and Haemostasis, 2020
Hemophilia A and B are inherited X‐linked disorders of hemostasis, associated with an increased bleeding tendency. Patients with severe hemophilia have undetectable clotting factor levels and experience spontaneous bleeds.
Fabienne Kloosterman, Anne‐Fleur Zwagemaker, Amal Abdi, Samantha Gouw, Giancarlo Castaman, Karin Fijnvandraat   +5 more
doaj   +1 more source

"Prevalence of inhibitors in a population of 1280 Hemophilia A patients in Iran " [PDF]

Acta Medica Iranica, 2003
Development of inhibitor to factor VIII is the most serious complication of hemophilia therapy. To determine the prevalence of inhibitors in Iran hemophilia A patients exposed to blood products, 1280 hemophilia A patients (age range 9 months-84 years ...
Sharifian R, Hosseini M, Safai R, Tugeh Gh, Ehsani AH, Lak M, Jazebi M   +6 more
doaj   +2 more sources

Hemophilia A Resulting in Severe Hyperesthesia Due to Extraparenchymal Spinal Cord Hemorrhage in a Young Golden Retriever Puppy

Veterinary Sciences, 2022
A ten-week-old male Golden retriever puppy was presented with severe hyperesthesia, mild neurological deficits and episcleral bleeding. Clotting times showed a normal prothrombin time (PT) and prolonged activated partial thromboplastin time (aPPT ...
Charlotte Lubbers, Martijn Beukers, Niklas Bergknut, Geert Paes   +3 more
doaj   +1 more source

Rebalancing Hemostasis: Fitusiran as a First-in-Class RNAi Therapy in Hemophilia A and B. [PDF]

Health Sci Rep
ABSTRACT Background and Aims Fitusiran is a first‐in‐class RNA interference (RNAi) therapy that lowers antithrombin (AT) to rebalance hemostasis in people with Hemophilia A or B, with or without inhibitors. Its recent FDA approval marks a major shift toward factor‐independent prophylaxis.
Rehman RU, Fatima R, Akilimali A.
europepmc   +2 more sources