Results 81 to 90 of about 21,171,014 (388)

Acquired Haemophilia Occurring in Association With Bullous Pemphigoid

JEADV Clinical Practice
Acquired haemophilia A (AHA) is a rare condition due to neutralizing antibodies against Factor VIII and can result in severe bleeding manifestations. The association of AHA with bullous pemphigoid (BP) is rare, most frequently observed in the elderly ...
Wen Yang Benjamin Ho, Zi Teng Chai, Haur Yueh Lee, Yen Loo Lim   +3 more
doaj   +1 more source

Haemophilia care in Iraq; a multi-centre study

Journal of the Pakistan Medical Association
Objectives: To evaluate the level of care available to haemophilia patients. Method: The descriptive, retrospective analytical study was conducted from December 15, 2020, to March 1, 2021, after approval from the Mustansiriyah University, Baghdad ...
Nidal Karim Al Rahal, Meaad Kadhum Hassan, Obeida Amir Abid, Azeezah Mohammed Mohsin   +3 more
doaj   +1 more source

Haemophilia care in Europe:a survey of 19 countries [PDF]

, 2011
In 2009, a questionnaire was circulated to 19 national haemophilia patient organizations in Europe affiliated to the European Haemophilia Consortium (EHC) and the World Federation of Hemophilia (WFH) to seek information about the organization of ...
Giangrande, P.L.F., Noone, D., O'Mahony, B., Prihodova, L.   +3 more
core   +1 more source

Case of acquired haemophilia a in Southeast Asia following COVID-19 vaccine [PDF]

, 2022
Lee Ai Vuen, Evelyn Aun Su-Yin, Ahlam Naila Kori, TM Shah   +3 more
openalex   +1 more source

The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab

Haemophilia, 2019
As a result of the new treatment paradigm that the haemophilia community will face with the availability of novel (non‐factor) therapies, an updated consensus on ITI recommendations and inhibitor management strategies is needed.
M. Carcao, C. Escuriola-Ettingshausen, E. Santagostino, J. Oldenburg, R. Liesner, B. Nolan, A. Bátorová, S. Haya, G. Young   +8 more
semanticscholar   +1 more source

A Novel Transcriptional Slippage Mechanism Rescues Dystrophin Expression from a DMD Frameshift Variant

Annals of Neurology, EarlyView.
Pathogenic DMD variants usually follow the reading‐frame rule: out‐of‐frame changes cause Duchenne muscular dystrophy, whereas in‐frame ones produce Becker muscular dystrophy (BMD). We report a 23‐year‐old man with BMD‐like weakness, calf hypertrophy, elevated creatine kinase, and dilated cardiomyopathy.
Hiroya Naruse, Jun Mitsui, Akatsuki Kubota, So Okubo, Shuichiro Mitsuchi, Kensho Sumi, Shuichi Tanifuji, Shogo Komaki, Asuka Kitamura, Meiko Maeda, Daiki Yashita, Atsushi Sudo, Takashi Matsukawa, Masashi Hamada, Wataru Satake, Shoji Tsuji, Tatsushi Toda   +16 more
wiley   +1 more source

The experiences of people with haemophilia and their families of gene therapy in a clinical trial setting: regaining control, the Exigency study

Orphanet Journal of Rare Diseases, 2022
Background Gene therapy has the potential to change the life experience of people with haemophilia and family members. Few studies have sought to explore the impact of gene therapy on both individuals and families.
Simon Fletcher, Kathryn Jenner, Luke Pembroke, Michael Holland, Kate Khair   +4 more
doaj   +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

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai, Shriarjun Shastry, Morgan R. Hurst, Arianna Minzoni, Eduardo Barbieri, Luke Remmler, Eugene N. Muratov, Michael A. Daniele, Stefano Menegatti, Alexander Tropsha   +9 more
wiley   +1 more source

Causes of death and mortality trends related to hemophilia in Brazil, 1999 to 2016

Hematology, Transfusion and Cell Therapy, 2021
Introduction: Multiple cause of death methodology enhances mortality studies beyond the traditional underlying cause of death approach. Aim: This study aims to describe causes of death and mortality issues related to haemophilia with the use of multiple ...
Augusto Hasiak Santo
doaj   +1 more source