Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A +5 more
core +1 more source
Mild hemophilia A (HA), defined by clinical features and factor VIII coagulant activity (FVIII:C) between 0.05 and 0.40 IU mL(-1), is characteristically distinct from severe HA. Indeed, although the molecular characterization of mild HA has permitted the identification of specific underlying mutations, its clinical phenotype is strikingly different ...
Franchini M +2 more
openaire +3 more sources
Establishment and Evolution of China National Hemophilia Registry
Hemophilia is an inherited bleeding disorder and a type of rare disease that is hereditary, lifelong and disabling. The establishment of a National Hemophilia Registry is foundational to treating hemophilia.
XUE Feng, YANG Renchi
doaj +1 more source
The history and evolution of the clinical effectiveness of haemophilia type a treatment: a systematic review. [PDF]
First evidence of cases of haemophilia dates from ancient Egypt, but it was when Queen Victoria from England in the 19th century transmitted this illness to her descendants, when it became known as the "royal disease".
A Aleem +48 more
core +2 more sources
"Prevalence of inhibitors in a population of 1280 Hemophilia A patients in Iran " [PDF]
Development of inhibitor to factor VIII is the most serious complication of hemophilia therapy. To determine the prevalence of inhibitors in Iran hemophilia A patients exposed to blood products, 1280 hemophilia A patients (age range 9 months-84 years ...
Sharifian R +6 more
doaj +2 more sources
Hemophilia management: Huge impact of a tiny difference
Hemophilia A and B are inherited X‐linked disorders of hemostasis, associated with an increased bleeding tendency. Patients with severe hemophilia have undetectable clotting factor levels and experience spontaneous bleeds.
Fabienne Kloosterman +5 more
doaj +1 more source
A ten-week-old male Golden retriever puppy was presented with severe hyperesthesia, mild neurological deficits and episcleral bleeding. Clotting times showed a normal prothrombin time (PT) and prolonged activated partial thromboplastin time (aPPT ...
Charlotte Lubbers +3 more
doaj +1 more source
Endothelial cells derived from patients' induced pluripotent stem cells for sustained factor VIII delivery and the treatment of hemophilia A. [PDF]
Hemophilia A (HA) is a bleeding disorder characterized by spontaneous and prolonged hemorrhage. The disease is caused by mutations in the coagulation factor 8 gene (F8) leading to factor VIII (FVIII) deficiency.
Agu, Emmanuel +9 more
core +1 more source
Utilization evaluation of factor concentration and frequencyof bleeds among patients with haemophilia "A" and haemophilia "B" in northwest Iran [PDF]
Background Haemophilia A and B are X-linked bleeding disorders which result in decreased blood levels of coagulants. According to some studies, Hemophilia Severity Score (HSS) is higher in severe Haemophilia A(HA) than in severe Haemophilia B(HB ...
Roya Dolatkhah +5 more
doaj +1 more source
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.
Pooja Sethi +3 more
openaire +4 more sources

