Progress towards gene therapy for haemophilia B [PDF]
, 2014 Nishil Patel +3 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
Optimal trough levels in haemophilia B: Raising expectations. [PDF]
Haemophilia, 2020 Walsh C, Coppens M, Escobar M, Wang M.
europepmc +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
Pregnancy outcomes in female carriers of haemophilia B Leyden [PDF]
, 2020 Jinal Gandhi +3 more
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
Continuous infusion factor replacement in haemophilia B during and after cardiac surgery: the better choice? [PDF]
BMJ Case Rep, 2020 Farid S, Sewaralthahab S, Smith HP.
europepmc +1 more source
Low‐factor consumption for major surgery in haemophilia B with long‐acting recombinant glyco
, 2016 Miguel A. Escobar +11 more
openalex +1 more source
Benign endobronchial lesion causing massive haemoptysis in a patient with haemophilia B. [PDF]
BMJ Case Rep, 2020 Mosalem O +3 more
europepmc +1 more source