Results 11 to 20 of about 2,583,576 (275)

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

Phase III dose selection of marzeptacog alfa (activated) informed by population pharmacokinetic modeling: A novel hemostatic drug

CPT: Pharmacometrics &Systems Pharmacology, Volume 11, Issue 12, Page 1628-1637, December 2022., 2022
Abstract Marzeptacog alfa (activated) (MarzAA) is an activated recombinant human FVII (rFVIIa) variant developed as subcutaneous (s.c.) administration for the treatment or prevention of bleeding episodes in patients with hemophilia A (HA) or hemophilia B (HB) with inhibitors and other rare bleeding disorders.
Alan Faraj, Tom Knudsen, Shraddha Desai, Linda Neuman, Grant E. Blouse, Ulrika S. H. Simonsson   +5 more
wiley   +1 more source

CRISPR applications for Duchenne muscular dystrophy: From animal models to potential therapies

WIREs Mechanisms of Disease, Volume 15, Issue 1, January/February 2023., 2023
CRISPR‐Cas9 gene‐editing technology enables the rapid generation of animal models for Duchenne muscular dystrophy research and has potential to be developed as CRISPR therapy for the long lasting genetic correction of causal mutations. Abstract CRISPR gene‐editing technology creates precise and permanent modifications to DNA.
Yu C. J. Chey, Jayshen Arudkumar, Annemieke Aartsma‐Rus, Fatwa Adikusuma, Paul Q. Thomas   +4 more
wiley   +1 more source

Efficacy and safety in patients with haemophilia A switching to octocog alfa (BAY 81–8973): Final results of the global real‐world study, TAURUS

European Journal of Haematology, Volume 110, Issue 1, Page 77-87, January 2023., 2023
Abstract Objectives To report the final results of the 2‐year TAURUS study, assessing weekly prophylaxis dosing regimens of octocog alfa (Kovaltry®/BAY 81–8973) used in standard clinical practice in patients with moderate‐to‐severe haemophilia A. Methods TAURUS (NCT02830477) is a phase 4, multinational, prospective, non‐interventional, single‐arm study
Cristina Santoro, Beng Fuh, Phu Quoc Le, Philip Maes, Rubén Berrueco, Eva Maria Mingot‐Castellano, Sylvia von Mackensen, Claudia Tueckmantel, Jose Francisco Cabre‐Marquez, Michael Wang   +9 more
wiley   +1 more source

Relation of bleeding patterns and factor VIII levels in children with hemophilia

Paediatrica Indonesiana, 2016
Background The high costs of factor VIII examination cause the delay in the diagnosis of hemophilia A; consequently many pa- tients do not receive adequate therapy which results in failure to survive into adulthood or survive with creeple.
Rina Rahardiani, H. S. Moeslichan MZ, Agus Firmansyah   +2 more
doaj   +1 more source

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

Establishment and Evolution of China National Hemophilia Registry

罕见病研究, 2022
Hemophilia is an inherited bleeding disorder and a type of rare disease that is hereditary, lifelong and disabling. The establishment of a National Hemophilia Registry is foundational to treating hemophilia.
XUE Feng, YANG Renchi
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  

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