Results 11 to 20 of about 118,583,499 (297)
Hemophilia in pediatric age [PDF]
Romanian Journal of Pediatrics, 2023 Hemophilia represents the most common inherited bleeding disorder linked to the X chromosome, which, if not properly treated, can lead to lifelong disabilities.
Cristina Elena Singer +7 more
doaj +1 more source
GENETIC DIAGNOSTICS OF HEMOPHILIA A [PDF]
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 +2 more
doaj +1 more source
HEMATURIA IN HEMOPHILIA: WHAT DO WE KNOW? A CHALLENGING CASE STUDY AND LITERATURE REVIEW [PDF]
Euromediterranean Biomedical Journal, 2022 Hemophilia is a genetically determined bleeding disorder, which, if not properly managed, can cause lifelong disabilities. Hemorrhages in the joints and soft tissues are largely studied.
Alessandra Di Nora
doaj +1 more source
Emicizumab-induced photosensitivity
TH Open, 2022 Emicizumab constitutes a novel and effective prophylaxis for hemophilia A patients with and without inhibitors. In this case report, we describe an emicizumab-induced photosensitivity that forced permanent sun-exposure suppression.
Pedro Asensi Cantó +6 more
doaj +1 more source
Clinical Pediatric Hematology-Oncology, 2022 Background : : Hemophilia requires a lifetime care for bleeding control and complications. Although patients diagnosed with hemophilia receive factor replacement, they also experience a variety of medical problems as they age.
Hyeun Su Seo +4 more
doaj +1 more source
Updates on Novel Non-Replacement Drugs for Hemophilia
Pharmaceuticals, 2022 Over the last decade, the world of hemophilia has experienced an unprecedented therapeutic advance, thanks to the progress in bioengineering technologies, leading to the introduction of drugs with novel mechanisms of action based on restoring thrombin ...
Roberta Gualtierotti +8 more
doaj +1 more source
Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy
Hepatology, EarlyView., 2022 Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann +25 more
wiley +1 more source
, 2022 Hemophilia is a group of hereditary hemorrhagic diseases. Hemorrhages mostly occur in musculoskeletal system and is the main cause for disability. Rehabilitation plays an important role in the comprehensive management in hemophilia.
Hemophilia Treatment Center Collaborative Network of China +1 more
core +1 more source
Research and Practice in Thrombosis and Haemostasis, 2021 Background Emicizumab, a bispecific antibody factor VIII mimetic, is approved for prophylaxis in hemophilia, and has different risks and side effects compared to factor VIII products.
Beth Boulden Warren +9 more
doaj +1 more source
Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A. [PDF]
, 2012 nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Tonn, Torsten +29 more
core +1 more source