Results 111 to 120 of about 48,006 (264)

Prophylaxis in Hemophilia

Clinical and Biomedical Research, 2023
Hemophilia is an inherited X-linked coagulopathy defined by a deficiency or abnormality in the clotting function of factor VIII (Hemophilia A) or factor IX (Hemophilia B).
Júlia Plentz Portich, Dayenne Catelli, Ebellins Tabares Calvache, Cristiane Seganfredo Weber, Daniel Sander Hoffmann, Guilherme Rasia Bosi, Leo Sekine, Lucia Mariano da Rocha Silla   +7 more
doaj  

Representation and Treatment of Haemofilia in the general populaton in Macedonia [PDF]

, 2012
Introduction: Defects in blood coagulation can be congenital or acquired. Haemophilia belongs to the congenital (hereditary) coagulopathies.It is inherited recessively by a mutant gene located on the X-chromosome, therefore it affects only men. Women are
Velickova, Nevenka
core  

Rare Bleeding Disorders and Bleeding Disorder of Unknown Cause: Current Understanding and Recent Developments

Haemophilia, EarlyView.
ABSTRACT Rare bleeding disorders (RBDs) represent a diverse group of inherited conditions involving coagulation factors or platelets. These conditions, such as Glanzmann thrombasthenia (GT) or severe coagulation factor deficiencies, are uncommon. In contrast, bleeding disorder of unknown cause (BDUC) is a diagnosis of exclusion without an identifiable ...
Alessandro Casini, Robert Klamroth, Bauke Haisma, Dino Mehic, Saskia E. M. Schols   +4 more
wiley   +1 more source

Final Analysis of the Phase 1/2 Trial of Valoctocogene Roxaparvovec for Severe Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Valoctocogene roxaparvovec is an adeno‐associated virus vector serotype 5 (AAV5)‐mediated gene therapy for severe haemophilia A (HA). Aim Report the final safety and efficacy results of the phase 1/2 trial of valoctocogene roxaparvovec. Methods An open‐label phase 1/2 trial (NCT02576795) enrolled adult males with severe HA (factor
Priyanka Raheja, Savita Rangarajan, Will Lester, Bella Madan, Glenn F. Pierce, Emily Symington, Carolyn Millar, Dane Osmond, Mingjin Li, Konstantia‐Maria Chavele   +9 more
wiley   +1 more source

Maximum Likelihood Estimation of the Multivariate Normal Mixture Model [PDF]

The Hessian of the multivariate normal mixture model is derived, and estimators of the information matrix are obtained, thus enabling consistent estimation of all parameters and their precisions.
Boldea, Otilia, Magnus, Jan R.
core   +1 more source

cGMP Recombinant FIX for IV and Oral Hemophilia B Therapy [PDF]

, 2005
Three specific aims are proposed: Specific Aim # 1. Process engineer and scale-up the recovery and purification of transgenic recombinant human Factor IX.
Dernell, William, KEY PERSON, Julian, Cooper, LEAD INVESTIGATOR, Manning, Mark, LEAD INVESTIGATOR, Meagher, Michael, LEAD INVESTIGATOR, Mohanan, Paul, LEAD INVESTIGATOR, Nichols, Timothy, LEAD INVESTIGATOR, Stephan, Abramson, LEAD INVESTIGATOR, Swaanson, Todd, KEY PERSON, Van Cott, Kevin E., Velander, William H., PRINCIPAL INVESTIGATOR   +9 more
core   +1 more source

Challenges and Opportunities in Post‐Marketing Reporting of Factor VIII Inhibitors With Efanesoctocog Alfa

Haemophilia, EarlyView.
Jennifer Dumont, Linda Bystricka, Graham Neill, Mahnouch Georget, Sriya Gunawardena, Elena Santagostino   +5 more
wiley   +1 more source

The Swiss Haemophilia Registry–Report From the First 8 Years

Haemophilia, EarlyView.
ABSTRACT Introduction Patient registries capture disease related information and provide a valuable source for real‐world data on rare diseases and their management. The Swiss Haemophilia Registry (SHR) was established in 2015 on the basis of a new Swiss federal human research act.
Alessandra Bosch, Lorenzo Alberio, Pierre Fontana, Lukas Graf, Johanna A. Kremer Hovinga, Nicolas von der Weid, Mattia Rizzi, Manuela Albisetti, the Swiss Haemophilia Network   +8 more
wiley   +1 more source

Sociodemographic and clinical characteristics of persons who experienced spontaneous hepatitis C viral clearance. [PDF]

, 2019
BackgroundIn the United States Hepatitis C virus (HCV) viral clearance is estimated to range between 20 and 30%. The objective of this study was to estimate the frequency of HCV clearance and identify correlates of viral clearance among patients newly ...
Baik, Yeonsoo, Chew, Kara W, He, Di, Javanbakht, Marjan, Kimble, Mabel Michille, Klausner, Jeffrey D, Ramirez, Samantha, Saab, Sammy, Stafylis, Chrysovalantis   +8 more
core  

The Value of Public Inquiries, Ethical Accountability, and Patient Voices: Reflections on the Infected Blood Inquiry

Haemophilia, EarlyView.
ABSTRACT Introduction This article contributes to the continuing conversation in Haemophilia about the UK Infected Blood Inquiry (IBI). Discussion within the journal to date has largely foregrounded professional and technical perspectives. Aim This article aims to bring back into view two elements central to the Inquiry—patient voice and the roles of ...
Richard Gorman, Clive Smith, Bobbie Farsides, Emma Cave   +3 more
wiley   +1 more source