Results 71 to 80 of about 82,032 (316)

A viable mouse model of factor X deficiency provides evidence for maternal transfer of factor X. [PDF]

, 2008
BackgroundActivated factor X (FXa) is a vitamin K-dependent serine protease that plays a pivotal role in blood coagulation by converting prothrombin to thrombin. There are no reports of humans with complete deficiency of FX, and knockout of murine F10 is
Arruda, VR, Chu, K, Golden, JA, Herzog, RW, High, KA, Labosky, PA, Margaritis, P, Tai, SJ   +7 more
core   +2 more sources

Gene Therapy and Gene Editing in Type 1 Diabetes: CRISPR‐Based β‐Cell Replacement and Treg Immune Modulation Approaches

Diabetes, Obesity and Metabolism, EarlyView.
ABSTRACT Background Type 1 diabetes mellitus (T1DM) is a chronic autoimmune disease marked by the destruction of pancreatic β‐cells, resulting in lifelong dependence on exogenous insulin. Despite advances in insulin delivery and glucose monitoring technologies, patients remain at risk for acute and long‐term complications, underscoring the need for ...
Tzu‐Min Lin, Hui‐Fang Chang, Tzu‐Ching Lin, Cheng‐Han Lin, Yu‐Ling Sun, Chih‐Sheng Lin   +5 more
wiley   +1 more source

Clinical perspectives of emerging pathogens in bleeding disorders. [PDF]

, 2006
As a result of immunological and nucleic-acid screening of plasma donations for transfusion-transmissible viruses, and the incorporation of viral reduction processes during plasma fractionation, coagulation-factor concentrates (CFC) are now judged safe ...
Bozzette, Samuel, Diamond, Michael, Koerper, Marion A, Kulkarni, Roshni, Ludlam, Christopher A, Powderly, William G, Ritchie, Bruce, Siegel, Jamie, Simmonds, Peter, Stanley, Samuel, Tapper, Michael L, von Depka, Mario   +11 more
core   +1 more source

Von Willebrand Disease [PDF]

, 2017
Von Willebrand Disease (VWD) is a blood clotting disorder characterized by low levels of the Von Willebrand Factor (VWF) in the blood. VWF is functions to bind platelets and promote clotting of the blood when vascular injury occurs.
Misla David, Ramon
core   +2 more sources

Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and Von Willebrand Disease

Haemophilia, EarlyView.
ABSTRACT Introduction The 2024 ISTH clinical practice guideline (CPG) for treatment of congenital haemophilia, the NBDF‐McMaster Guideline on Care Models for Haemophilia Management, and ASH ISTH NBDF WFH guidelines on the diagnosis and management of VWD all utilised GRADE methodology.
Mark W. Skinner, Manuela Albisetti, Jesús Ardila, Jan Astermark, Jan Blatny, Manuel Carcao, Pratima Chowdary, Nathan T. Connell, Miguel Crato, Yesim Dargaud, Roseline d'Oiron, Amy L. Dunn, Miguel A. Escobar, Carmen Escuriola‐Ettingshausen, Emna Gouider, Annie Harroche, Cedric Hermans, Victor Jimenez‐Yuste, Radoslaw Kaczmarek, Gili Kenet, Liane Khoo, Robert Klamroth, Florian Langer, David Lillicrap, Johnny Mahlangu, Christoph Male, Tadashi Matsushita, Sandrine Meunier, Wolfgang Miesbach, Beatrice Nolan, Johannes Oldenburg, Brian O'Mahony, Margareth Ozelo, Glenn F. Pierce, Gloria Ramos, Michael Recht, Olivia Romero‐Lux, Dawn Rotellini, Rita C. Santoro, Tammuella C. Singleton, Alok Srivastava, Sophie Susen, Kate Talks, Huyen Tran, Leonard A. Valentino, Jerzy Windyga, Renchi Yang, Maria Elisa Mancuso, also on behalf of AICE, AHAD‐AP, AHCDC, ATHN, CLAHT, EHC, French CRH & MHEMO, HTCCNC, NBDF, PEDNET, UKHCDO, WFH   +59 more
wiley   +1 more source

Safety and Efficacy of Damoctocog Alfa Pegol Prophylaxis in Patients With Severe Haemophilia A: Results of an Interventional, Post‐Marketing Study

Haemophilia, EarlyView.
ABSTRACT This is a plain language summary of the results of a trial of damoctocog alfa pegol (BAY 94–9027, Jivi).
Pål André Holme, Lone Hvitfeldt Poulsen, Claudia Tueckmantel, Monika Maas Enriquez, María Teresa Alvarez Román, Raimondo De Cristofaro   +5 more
wiley   +1 more source

Factor 8 Gene Mutation Spectrum of 270 Patients with Hemophilia A: Identification of 36 Novel Mutations

Turkish Journal of Hematology, 2020
Objective: Hemophilia A (HA) is the most severe X-linked inherited bleeding disorder caused by hemizygous mutations in the factor 8 (F8) gene. The aim of this study is to determine the mutation spectrum of the F8 gene in a large HA cohort from Turkey ...
Tahir Atik, Esra Işık, Hüseyin Onay, Bilçağ Akgün, Moharram Shamsali, Kaan Kavaklo, Melike Evim, Gülen Tüysüz, Namık Yaşar Özbek, Fahri Şahin, Zafer Salcıoğlu, Canan Albayrak, Yeşim Oymak, Ekrem Ünal, Fatma Burcu Belen, Ebru Yılmaz Keskin, Can Balkan, Birol Baytan, Alphan Küpesiz, Vildan Culha, Tuba Nur Tahtakesen, Adalet Meral Güneş, Ferda Özkınay   +22 more
doaj   +1 more source

Heavy hematuria requiring cystectomy in a patient with hemophilia A: a case report and literature review [PDF]

, 2015
BACKGROUND: Hemophilia A is an X-linked recessive disorder caused by a deficiency in factor VIII. Hemophilia A affects 1 in 5,000–10,000 males. Hematuria is frequent in hemophilia.
Kimitoshi Saito, Masaru Hirai, Satoshi Washino, Tomoaki Miyagawa, Yutaka Kobayashi   +4 more
core   +1 more source

Central Nervous System Bleeding in Children With Haemophilia in Limited Resource

Haemophilia, EarlyView.
ABSTRACT Introduction Central nervous system (CNS) bleeding in children with haemophilia is a life‐threatening complication that may cause severe neurological sequelae or death. In resource‐limited settings, where prophylaxis is not universally accessible, its patterns and outcomes may differ from those in high‐income countries.
Patcharee Komvilaisak, Sudarat Sirichaipornsak, Paiporn Sripraya, Napat Laoaroon, Kunanya Suwannaying, Pichayen Duanthongphon   +5 more
wiley   +1 more source

Enhancing the Evidence for Care in Underserved Bleeding Disorders Communities

Haemophilia, EarlyView.
ABSTRACT Background Major advances in haemophilia care have not translated equitably across all populations. Individuals with rare bleeding disorders (RBDs), people living in low‐ and lower‐middle‐income countries (LMICs) and women and girls with inherited bleeding disorders (WGWBD) continue to face significant diagnostic, therapeutic and research ...
Johnny Mahlangu
wiley   +1 more source