Results 151 to 160 of about 13,000,271 (286)
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 +7 more
wiley +1 more source
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
Muscle-Directed Gene Transfer and Transient Immune Suppression Result in Sustained Partial Correction of Canine Hemophilia B Caused by a Null Mutation [PDF]
, 2001 Roland W. Herzog +4 more
openalex +1 more source
Impact of Model‐Informed Drug Development on Drug Development Cycle Times and Clinical Trial Cost
Clinical Pharmacology &Therapeutics, Volume 118, Issue 2, Page 378-385, August 2025.Model‐informed drug development (MIDD) integrates data to quantify benefit/risk informing objective drug discovery and development decisions. An additional critical benefit of MIDD is postulated to be improvement in trial and program efficiencies. While the application of MIDD has grown, there have been no clear examples across programs to demonstrate ...
Vaishali Sahasrabudhe +4 more
wiley +1 more source
Sustained and Complete Phenotype Correction of Hemophilia B Mice Following Intramuscular Injection of AAV1 Serotype Vectors [PDF]
, 2001 Hengjun Chao +4 more
openalex +1 more source
Association Between Hair Cortisol and Psychopathology in Children With a Chronic Physical Illness
Stress and Health, Volume 41, Issue 4, August 2025.ABSTRACT Children with a chronic physical illness (CPI) experience significant stress and are at a greater risk of psychopathology. However, little is known about chronic stress and its relationship with psychopathology in this population. Over the last decade, hair cortisol concentration (HCC) has emerged as a viable biomarker of chronic stress.
Emma A. L. Littler +3 more
wiley +1 more source
250. High Level Expression of Canine Factor IX in Hemophilia B Dogs after Readminstration of AAV2/5 Vectors [PDF]
, 2002 openalex +1 more source
The UK Infected Blood Inquiry: A Personal Reflection
Haemophilia, EarlyView.
Peter R. Foster
wiley +1 more source
European Journal of Haematology, Volume 115, Issue 2, Page 134-141, August 2025.ABSTRACT Objective To report data from an ITI chart review study (NCT03951103) for first‐time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A. Methods Retrospective and prospective real‐world data are reported from a non‐interventional, multicentre study of patients who had been or were currently ...
Robert Klamroth +7 more
wiley +1 more source