Results 51 to 60 of about 9,259 (230)

Haemostatic action of a topical foam-based patch (VELSEAL-T) in haemophiliac patients with external bleeding

The Journal of Haemophilia Practice, 2020
Haemophilia is an X-linked congenital bleeding disorder due to deficiency of coagulation factor VIII (in haemophilia A) or factor IX (in haemophilia B) caused by mutations of the respective clotting factor genes.
Dutta Anupam, Dutta Taniya Sarkar, Das Anup Kumar, Dey Pranoy   +3 more
doaj   +1 more source

Ddavp treatment in children with haemophilia b [PDF]

British Journal of Haematology, 2001
WOS ...
Aydinok, Y, Kavakli, K, Karapinar, D, Balkan, C   +3 more
openaire   +3 more sources

Increased Thrombogenicity is Associated With Coronary Microvascular Dysfunction in Patients With STEMI—A Proof‐of‐Concept Study

Catheterization and Cardiovascular Interventions, EarlyView.
ABSTRACT Background Up to 50% of patients with ST‐segment elevation myocardial infarction (STEMI) undergoing primary percutaneous coronary intervention (pPCI) develop coronary microvascular dysfunction (CMD). Aims This study aims to assess whether a prothrombotic state of coronary blood, defined by thromboelastography (TEG), is associated with post ...
Roberto Scarsini, Denis Leonardi, Andrea Bottardi, Concetta Mammone, Leonardo Portolan, Caterina Butturini, Verdiana Galli, Francesco Della Mora, Alessandro Ruzzarin, Sara Pazzi, Anna Piccoli, Simone Fezzi, Domenico Tavella, Gabriele Pesarini, Leonardo Gottin, Flavio Ribichini   +15 more
wiley   +1 more source

Model‐Informed Drug Development Applications and Opportunities in mRNA‐LNP Therapeutics

Clinical Pharmacology &Therapeutics, EarlyView.
The utilization of lipid nanoparticles (LNP) for encapsulating mRNA has revolutionized the field of therapeutics, enabling the rapid development of COVID‐19 vaccines and cancer vaccines. However, the clinical development of mRNA‐LNP therapeutics faces numerous challenges due to their complex mechanisms of action and limited clinical experience.
Jiawei Zhou, Rohit Rao, Monica E. Shapiro, Nessy Tania, Cody Herron, Cynthia J. Musante, Jim H. Hughes   +6 more
wiley   +1 more source

Relationship between Dose, Factor IX Activity Levels and Bleeding Probability for rIX‐FP Prophylaxis in Hemophilia B: A Repeated Time‐to‐Event Analysis

Clinical Pharmacology &Therapeutics, EarlyView.
In hemophilia B, pharmacokinetic (PK)‐guided dosing of extended half‐life factor IX (EHL‐FIX) concentrates can secure targeted FIX exposure. Target FIX activity levels in plasma should be individually set primarily taking bleeding tendency into account, alongside the presence of target joints, physical activity, and preferred dosing schedules. In other
Sjoerd F. Koopman, Marjon H. Cnossen, Ron A.A. Mathot, on behalf of the OPTI‐CLOT study group and SYMPHONY consortium, S. H. Reitsma   +4 more
wiley   +1 more source

Optimizing Patient Registries for Regulatory Decision Making ‐ Key Learnings From an HMA/EMA Multistakeholder Workshop

Clinical Pharmacology &Therapeutics, EarlyView.
The Joint Heads of Medicines Agencies and European Medicines Agency's (HMA/EMA) big data initiative paves the way for better integration of real‐world data, including data from patient registries, into regulatory decisions on medicines. This article focuses on the outcome of a two‐day multistakeholder workshop organized by EMA in 2024, which explored ...
Kelly Plueschke, Carla Jonker, Hanna Kankanen, Thorsten Vetter, Bruno Sepodes, Lutz Naehrlich, Jan Hillert, Gracy Crane, Sabine Straus, Paolo Foggi, Simona Martin, Christina Kyriakopoulou, Peter Mol, Francesca Day, Kieran Breen, Neil Bennett, Mencia de Lemus Belmonte, Simon Bennett, Patrice Verpillat, Kit C. B. Roes, Ana Cochino, Franz Schaefer, Jesús María Hernández‐Rivas, Patricia McGettigan, Peter Arlett   +24 more
wiley   +1 more source

Difference in Fibrin Clot Structure in Haemophilia A and Haemophilia B

Blood, 2016
Abstract Introduction: Hemophilia A and Hemophilia B are X-linked bleeding disorders characterized by deficiency of coagulation factor VIII and IX respectively. The disorders have traditionally been considered to display identical symptoms.
Jovan P. Antovic, Roza Chaireti, Margareta Holmström, Vincent Luong, Nida Mahmoud Hourani Soutari, Maria Berndtsson   +5 more
openaire   +2 more sources

Quantifying Critical Quality Attributes of Protein Therapeutics by Sodium Dodecyl Sulfate–Capillary Gel Electrophoresis With Native Fluorescence Detection

ELECTROPHORESIS, EarlyView.
ABSTRACT In the biopharmaceutical industry, the sodium dodecyl sulfate–capillary gel electrophoresis (SDS–CGE) assay is often used to assess therapeutic critical quality attributes (CQAs). Traditional detection methods for SDS–CGE methods, such as ultraviolet (UV) absorbance and laser‐induced fluorescence (LIF), are widely used but come with ...
Zaifang Zhu
wiley   +1 more source

Exploring the Potential and Advancements of Circular RNA Therapeutics

Exploration, EarlyView.
Given the remarkable advantages in terms of stability, sustained expression profile, safety, wide range of druggable targets, scalable and cost‐effective manufacturing capabilities, circRNA is currently undergoing intensive investigation for various therapeutic applications such as vaccines, protein replacement, genetic disease treatment, gene therapy,
Lei Wang, Lianqing Wang, Chunbo Dong, Jialong Liu, Guoxin Cui, Shan Gao, Zhida Liu   +6 more
wiley   +1 more source

Distribution and predictors of haemophilia-related costs in the United Kingdom: analysis of the CHESS I and CHESS II burden of illness studies

BMC Health Services Research
Background Few studies have evaluated direct medical or societal costs of haemophilia in the United Kingdom (UK), and how patient characteristics impact future costs is uncertain.
Ione Woollacott, Amit Chhabra, Tom Burke, Jack Brownrigg, Lucy Richardson, Enrico Ferri Grazzi, Jamie O’Hara, Josie Godfrey, Michael Laffan   +8 more
doaj   +1 more source