Results 71 to 80 of about 68,267 (269)

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

Comparative Investigation of Thromboelastometry and Thrombin Generation for Patients Receiving Direct Oral Anticoagulants or Vitamin K Antagonists

Diagnostics
Background. Alterations induced by direct oral anticoagulants (DOACs) or vitamin K antagonists (VKAs) to thromboelastometry and thrombin generation are not well defined.
Armando Tripodi, Marco Capecchi, Erica Scalambrino, Marigrazia Clerici, Barbara Scimeca, Pasquale Agosti, Paolo Bucciarelli, Andrea Artoni, Flora Peyvandi   +8 more
doaj   +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

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

Emergency department visits in children with hemophilia [PDF]

, 2012
Background The pediatric emergency department (ED) management of bleeding and other complications of hemophilia constitutes an increasingly important component of hemophilia therapy.
Callaghan, Michael U., Chitlur, Meera, Lusher, Jeanne M., Rajpurkar, Madhvi, Zia, Ayesha, Özgönenel, Bülent   +5 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

On a break with the X: the role of repair of double-stranded DNA breaks in X-linked disease [PDF]

, 2012
The problem of managing free reactive DNA ends in eukaryotic cells has resulted in the development of a number of mechanisms in order to ensure that free ends are rendered non-reactive, or that the double-strand DNA breaks generating the free ends are ...
Cecceroni, Lucia, De Caris, Laura, Tummala, Hemanth   +2 more
core   +4 more sources

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

Haemophilia and joint disease: pathophysiology, evaluation and management [PDF]

, 2011
In patients with haemophilia, regular replacement therapy with clotting factor concentrates (prophylaxis) is effective in preventing recurrent bleeding episodes into joints and muscles.
Berntorp, Erik, Knobe, Karin
core   +3 more sources