Results 81 to 90 of about 48,006 (264)

FVIIIa Mimetics: New Approaches and Next‐Generation Initiatives

Haemophilia, EarlyView.
ABSTRACT Emicizumab has revolutionized hemophilia A care, yet limitations regarding the “ceiling” of hemostatic efficacy (equivalent to mild hemophilia) and global access persist. This review critically examines two distinct paradigms shaping the future of care: Innovation and Access. Regarding innovation, we synthesize the latest clinical data on next‐
Tadashi Matsushita, Keiji Nogami, Chandrakala Shanmukhaiah   +2 more
wiley   +1 more source

A Psychometric Analysis of the Haemophilia Quality of Life Questionnaire for Adults Physical Health Subscale and Total Scores: Data From the Phase 3 XTEND‐1 Trial in Patients With Severe Haemophilia A

Haemophilia, EarlyView.
ABSTRACT Introduction Bleeding episodes can impair physical functioning and cause chronic pain in haemophilia A. The Haemophilia Quality of Life Questionnaire for Adults (Haem‐A‐QoL) was administered in XTEND‐1; the Physical Health (PH) subscale evaluated Physical Health/Functioning. Aim To evaluate psychometric properties of Haem‐A‐QoL PH and estimate
Jérôme Msihid, Alfonso Iorio, Amanda Wilson, Nana Kragh, Lauren Nelson, Shanshan Qin, Annemieke Willemze   +6 more
wiley   +1 more source

Mild-severe hemophilia B impacts relationships of US adults and children with hemophilia B and their families: results from the B-HERO-S study

Patient Related Outcome Measures, 2019
Susan Cutter,1 Christine Guelcher,2 Susan Hunter,3 Dawn Rotellini,4 Spencer Dunn,5 David L Cooper61Penn Comprehensive Hemophilia and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA, USA; 2Comprehensive Hemostasis and ...
Cutter S, Guelcher C, Hunter S, Rotellini D, Dunn S, Cooper DL   +5 more
doaj  

Haemophilia and joint disease: pathophysiology, evaluation and management [PDF]

, 2011
In patients with haemophilia, regular replacement therapy with clotting factor concentrates (prophylaxis) is effective in preventing recurrent bleeding episodes into joints and muscles.
Berntorp, Erik, Knobe, Karin
core   +3 more sources

Predicting Recovery After Joint Bleeding in Persons With Bleeding Disorders

Haemophilia, EarlyView.
ABSTRACT Introduction Joint bleeds are burdensome and recovery differs from bleed to bleed. Identifying predictors of recovery could enable personalized treatment and monitoring; aiming to prevent long‐term joint deterioration, and facilitate faster return to activities. Aim To identify predictors of time to recovery after joint bleeding in people with
Gijs Aertssen, Lize F. D. van Vulpen, Wouter Foppen, Flora H. P. van Leeuwen, Merel A. Timmer   +4 more
wiley   +1 more source

Von Willebrand Disease [PDF]

, 2017
Von Willebrand Disease (VWD) is a blood clotting disorder characterized by low levels of the Von Willebrand Factor (VWF) in the blood. VWF is functions to bind platelets and promote clotting of the blood when vascular injury occurs.
Misla David, Ramon
core   +2 more sources

Gene Editing for Haemophilia—The Next Frontier

Haemophilia, EarlyView.
ABSTRACT The recently approved haemophilia A and B gene therapies via adeno‐associated virus (AAV) showed a promising therapeutic response after a single injection, but there are still limitations, including the potential loss of transgene expression and restriction in adults.
Mirko Pinotti, Gregory A. Newby, Sundar Selvaraj, Steven W. Pipe   +3 more
wiley   +1 more source

Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

Journal of Translational Medicine, 2017
Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia.
David M. Markusic, Timothy C. Nichols, Elizabeth P. Merricks, Brett Palaschak, Irene Zolotukhin, Damien Marsic, Sergei Zolotukhin, Arun Srivastava, Roland W. Herzog   +8 more
doaj   +1 more source

Inherited Bleeding Disorders in Pregnancy: Obstetric Management and Outcomes From a Tertiary Care Centre

Haemophilia, EarlyView.
ABSTRACT Objective This study aimed to evaluate the obstetric management, complications and clinical characteristics of pregnant women diagnosed with hereditary coagulation factor deficiencies at a tertiary obstetric centre over a 10‐year period. Methods We retrospectively reviewed a total of 19 pregnancies in 17 women with hereditary coagulation ...
Melis Altug Inan, Bilge Kapudere, Hanne Bulat Cim, Abdulkadir Turgut   +3 more
wiley   +1 more source

CO27 | Intracerebral hemorrhage in hemophilia A and B: a 10-year prospective multicenter study in Italy (2012–2022)

Bleeding, Thrombosis and Vascular Biology
Background and Aims: Intracerebral hemorrhage (ICH) is a severe and life-threatening complication in patients with hemophilia, often resulting in high mortality and long-term neurological impairment.
doaj   +1 more source