Results 31 to 40 of about 32,279 (169)

Hemophilia gene therapy knowledge and perceptions: Results of an international survey

Research and Practice in Thrombosis and Haemostasis, 2020
Background Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion.
Flora Peyvandi, David Lillicrap, Johnny Mahlangu, Claire McLintock, K. John Pasi, Steven W. Pipe, Wendy Scales, Alok Srivastava, Thierry VandenDriessche   +8 more
doaj   +1 more source

Thromboelastography in pre-surgery monitoring in Hemophilia A with high inhibitor titer: case report and literature review

Romanian Journal of Laboratory Medicine, 2020
The development of factor VIII inhibitors (allo-antibodies) continues to be a major complication in the management of severe forms of hemophilia A, especially as far as treatment and treatment response monitoring is concerned.
Melen Brinza, Uscatescu Valentina, Gheorghe Georgiana, Chiriac Elisabeta, Ciobanu Claudia, Orban Horia, Coriu Daniel   +6 more
doaj   +1 more source

Occurrence of FVIII Inhibitors in Hemophilia A Patients Following an Institutional Switch to a Third Generation B-Domain-Deleted FVIII

Clinical and Applied Thrombosis/Hemostasis, 2023
In 2018, Refacto AF R , a B-domain-deleted third-generation FVIII concentrate, became our preferential product. After the introduction, the development of inhibitors was prospectively monitored; retrospectively, we sought for risk factors in the patients
Louise H Hooimeijer MD, Marjet A Stein-Wit MD, Marja AJ Voskuilen RN, Michaël V Lukens PhD, Karina Meijer MD, PhD, Anja BU Mäkelburg MD, PhD, Rienk YJ Tamminga MD, PhD   +6 more
doaj   +1 more source

Hemophilia of orbit

Oman Journal of Ophthalmology, 2009
Hemophilic pseudotumor is an uncommon complication of factor VIII and IX deficiencies in the coagulation cascade and occurs in a wide spectrum of bones and soft tissues. We present a six-year-old boy with hemophilic pseudotumor localized in the right orbit.
Gunaseelan Karunanithi, Pooja Sethi, Sathyanarayana K Reddy, Vivekanandam   +3 more
openaire   +3 more sources

Hemophilia treatment and transfusion medicine

Zhongguo shuxue zazhi
The improvement of hemophilia treatment is attributed to the development of transfusion medicine, and the discovery of cryoprecipitate has opened up a new era of hemophilia treatment, laying the foundation for the development of modern hemophilia ...
FANG Yunhai, CHENG Yan, ZHANG Xinsheng
doaj   +1 more source

Seroprevalence to adeno‐associated virus type 6 in people with hemophilia B from a UK adult cohort

Research and Practice in Thrombosis and Haemostasis, 2022
Background Gene therapy shows promise as a potential “cure” for hemophilia A and B. Adeno‐associated virus (AAV) vectors are the leading platform to deliver modified genetic code of factor VIII or IX to the liver effecting endogenous production.
Sara Boyce, Izabela James, Savita Rangarajan, Nicola Curry, Catherine Bagot, Steven Austin, Mike Laffan, Sarah Mangles, Kandiah Chandrakumaran, Carina Mundy   +9 more
doaj   +1 more source

An international registry of patients with plasminogen deficiency (HISTORY)

Haematologica, 2020
Plasminogen deficiency is an ultra-rare multisystem disorder characterized by the development of fibrin-rich pseudomembranes on mucous membranes. Ligneous conjunctivitis, which can result in vision impairment or loss, is the most frequent symptom ...
Amy D. Shapiro, Marzia Menegatti, Roberta Palla, Marco Boscarino, Christopher Roberson, Paolo Lanzi, Joel Bowen, Charles Nakar, Isaac A. Janson, Flora Peyvandi   +9 more
doaj   +1 more source

Practices and challenges for hemophilia management under resource constraints in Thailand

Orphanet Journal of Rare Diseases, 2023
Hemophilia is an inherited bleeding disorder caused by deficiency of a specific coagulation factor. Factor VIII deficiency is responsible for hemophilia A while factor IX deficiency is responsible for hemophilia B. As per the 2020 annual global survey by
Chatphatai Moonla, Darintr Sosothikul, Bunchoo Pongtanakul, Bundarika Suwanawiboon, Chanchai Traivaree, Rungrote Natesirinilkul, Nongnuch Sirachainan, Pantep Angchaisuksiri   +7 more
doaj   +1 more source

Comparative Investigation of Thromboelastometry and Thrombin Generation for Patients Receiving Direct Oral Anticoagulants or Vitamin K Antagonists

Diagnostics
Background. Alterations induced by direct oral anticoagulants (DOACs) or vitamin K antagonists (VKAs) to thromboelastometry and thrombin generation are not well defined.
Armando Tripodi, Marco Capecchi, Erica Scalambrino, Marigrazia Clerici, Barbara Scimeca, Pasquale Agosti, Paolo Bucciarelli, Andrea Artoni, Flora Peyvandi   +8 more
doaj   +1 more source

GENETIC DIAGNOSTICS OF HEMOPHILIA A

Zdravniški Vestnik, 2004
Background. Hemophilia A is a chromosome X-linked bleeding disorder due to mutations in the FVIII gene. There are 163 hemophilia A patients in Slovene registry for Hemophilia.
Maruša Debeljak, Majda Benedik Dolničar, Lana Strmecki   +2 more
doaj