Results 131 to 140 of about 9,259 (230)

Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia B. WFH State‐of‐the‐Art Session on Therapeutic Gene Transfer Buenos Aires, Argentina [PDF]

, 2010
RR Montgomery, Paul E. Monahan, Margareth C. Ozelo   +2 more
openalex   +1 more source

Understanding supply sustainability of plasma‐derived medicinal products: Drivers and consequences of shortages

Vox Sanguinis, EarlyView.
Abstract Plasma‐derived medicinal products (PDMPs), particularly immunoglobulins (Igs), are essential treatments for numerous diseases, often serving as the primary therapeutic option and playing a critical role in patient care. The human origin of these products, however, can lead to supply constraints due to a lack of plasma collection, market ...
Miriam Belmonte, Anna Albiero, Filip Callewaert, Julien Patris, Amanda Whittal   +4 more
wiley   +1 more source

“I’d go on a hike with my kids… that would make me feel so happy.” Recognising what people with haemophilia B identify as meaningful when considering personal goals and the potential for gene therapy

The Journal of Haemophilia Practice
Historically people with haemophilia B (PwHB) have experienced adverse clinical outcomes including joint bleeds, pain and arthropathy. The current management of severe and moderate haemophilia B in the UK is largely through the use of factor replacement ...
Khair Kate, Nagra Ranjit, Gorman Richard, Fletcher Simon, Jenner Kathryn, Trim Joe, Collings Shuk-Li   +6 more
doaj   +1 more source

Prevalence of clinical hip abnormalities in haemophilia A and B: an analysis of the UDC database [PDF]

, 2012
Derek M. Kelly, Q. C. Zhang, J. Michael Soucie, Marilyn J. Manco‐Johnson, Donna DiMichele   +4 more
openalex   +1 more source

Development and Validation of a Liquid Chromatography High‐Resolution Mass Spectrometry Method for Blood Desmopressin Quantification and Its Application in Hemophilia A Patients

Rapid Communications in Mass Spectrometry, Volume 39, Issue 18, 30 September 2025.
ABSTRACT Desmopressin (DDAVP), which indirectly increases Coagulation Factor VIII concentrations in the blood, is a common treatment for bleeding disorders such as von Willebrand disease or hemophilia A. However, DDAVP exhibits significant variability in response due to interindividual differences in pharmacokinetics.
Sophie Hodin, Jean Escal, Catherine Feliu, Adeline Pontis, Laurie Josset, Adrian Serban, Benoît Guillet, Xavier Delavenne   +7 more
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

Medical conditions associated with Prolonged Activated Partial Thromboplastin time in Swat

Gomal Journal of Medical Sciences, 2010
Background: APPT is a commonly requested laboratory test in various bleeding conditions and as a screening test preoperatively. It is also used to monitor heparin therapy.
Mukamil Shah, Imran ud Din Khattak, Sania Tanweer Khattak, Ahad Abdul, Muhammad Amin Jan Mahsud   +4 more
doaj  

PSY40 Health-Related Quality of Life and Annual Direct Medical Cost of Patients With Haemophilia B in France: The EQOFIX Study [PDF]

, 2012
Benoı̂t Polack, Thierry Calvez, S. Claeyssens, Hérvè Chambost, A Derlon, Jenny Goudemand, C. Rothschild, Françoise Maurel, Marie-Christine Woronoff-Lemsi   +8 more
openalex   +1 more source

Joint health and treatment modalities in Nordic patients with moderate haemophilia A and B – The MoHem study [PDF]

, 2020
Ragnhild J. Måseide, Erik Berntorp, Jan Astermark, Anna Olsson, Maria Bruzelius, Tony Frisk, Vuokko Nummi, Riitta Lassila, Geir E. Tjønnfjord, Pål André Holme   +9 more
openalex   +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