Results 161 to 170 of about 73,362 (297)

Isolation and Properties of the Abnormal Factor IX Molecule of Hemophilia BM [PDF]

, 1977
Bjarne Østerud, K K Lavine, Carol K. Kasper, Samuel I. Rapaport   +3 more
openalex   +1 more source

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

Clinical Pharmacology &Therapeutics, Volume 118, Issue 3, Page 551-560, September 2025.
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

Nonneutralizing antibodies in Nordic persons with moderate hemophilia A and B (the MoHem study)

Research and Practice in Thrombosis and Haemostasis
Background: The impact of nonneutralizing antibodies (NNAs) in moderate hemophilia is elusive. Objectives: To explore the presence of NNAs in Nordic persons with moderate hemophilia A (MHA) and B (MHB) in relation to treatment modality, clinical outcome,
Ragnhild J. Måseide, MD, PhD, Erik Berntorp, MD, PhD, Jan Astermark, MD, PhD, Anna Olsson, MD, PhD, Maria Bruzelius, MD, PhD, Tony Frisk, MD, PhD, Vuokko Nummi, MD, PhD, Riitta Lassila, MD, PhD, Karin Strandberg, MD, PhD, Geir E. Tjønnfjord, MD, PhD, Pål A. Holme, MD, PhD   +10 more
doaj  

The UK Infected Blood Inquiry: A Personal Reflection

Haemophilia, EarlyView.
Peter R. Foster
wiley   +1 more source

Effectiveness of Pyridoxal‐5′‐Phosphate in PNPO Deficiency: A Systematic Review

Journal of Inherited Metabolic Disease, Volume 48, Issue 5, September 2025.
ABSTRACT Pyridox(am)ine 5′‐phosphate oxidase (PNPO) deficiency is an ultrarare inherited neurometabolic disease, characterized by primarily neonatal‐onset B6‐responsive epileptic encephalopathies. Treatment often requires sustainable access to high‐quality pyridoxal‐5′‐phosphate (PLP, i.e., active vitamin B6), although some patients (also) respond to ...
Nina N. Stolwijk, Laura van Dussen, Niels D. Reijnhout, Marion M. M. G. Brands, Bregje Jaeger, Peter T. Clayton, Carla E. M. Hollak, Annet M. Bosch   +7 more
wiley   +1 more source

Halfspace Depth

WIREs Computational Statistics, Volume 17, Issue 3, September 2025.
The halfspace depth generalizes quantiles to multivariate data. This is a bagplot—a depth‐based analog of a boxplot. It succinctly captures the geometry of the bivariate dataset (blue/red points) and identifies the four red points in the top left corner as deviating from the general pattern of the data.
Stanislav Nagy
wiley   +1 more source

Recent Advances in mRNA Delivery Systems for Cancer Therapy

Advanced Science, Volume 12, Issue 29, August 7, 2025.
This review systematically investigates the applications of mRNA therapy in cancer treatment, with particular emphasis on nonviral delivery systems, targeting strategies, stimulus‐responsive systems, and local delivery methods. Concluding with a meticulous evaluation, the review sheds light on the prevailing challenges while illuminating promising ...
Zheng Zhang, Ya‐Nan Fan, Si‐Qi Jiang, Ya‐Jing Ma, Yao‐Ru Yu, Yu‐Xin Qing, Qian‐Ru Li, Yi‐Lin Liu, Song Shen, Jun Wang   +9 more
wiley   +1 more source

The Role of the Consumer-Patient in the Growth of a Comprehensive Hemophilia Care Unit [PDF]

, 1977
Dorothy Child, John Kopicki, David VanHarlingen   +2 more
openalex   +1 more source

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

Catheterization and Cardiovascular Interventions, Volume 106, Issue 2, Page 836-845, August 1, 2025.
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

Hereditary Hemorrhagic Disorders Excluding Hemophilia Group

, 1963
Mitsuyasu Kagami
openalex   +2 more sources