SINCE the discovery of hemophilia B (PTC deficiency, congenital factor IX deficiency, Christmas disease) in 1952, 1–3 many attempts have heen made to prepare practical and effective concentrates ...

Grifols is working to enhance the lives of the roughly 100,000 people around the world who have hemophilia but receive little or no treatment. The company is halfway into an eight-year initiative ...

"Multiyear Factor VIII Expression After AAV Gene Transfer for Hemophilia A," New England Journal of Medicine, November 18, 2021, DOI: 10.1056/NEJMoa2104205 ...

Everyone experiences nosebleeds at some point, but nosebleeds with no known cause can be a sign of hemophilia, according to ...

The trial infuses SPK-8011, a new recombinant AAV vector engineered to express factor VIII in human liver cells, in 18 men with hemophilia A and demonstrates a 91.5% decrease in bleeding episodes.

A decline in annualized rates of factor VIII use of 98.6% and treated bleeding of 83.8% (P <.001 for both comparisons) A median factor VIII activity level of 5 IU/dL or higher in 88.1% of participants ...

Switching to extended half-life therapies for hemophilia enhances treatment adherence and reduces infusion frequency, despite ...

Hemophilia B, in which patients have low levels of clotting factor IX (FIX), has been used as a model disorder for the development of gene therapy research for more than two decades.

Send comments and news tips to [email protected]. Cite this: Gene Transfer Restores Clotting Factor Level in Hemophilia B - Medscape - Dec 13, 2011.

Hemophilia A results from genetic variations to the F8 gene on a person’s X chromosome. The F8 gene directs the production of clotting factor VIII, one of many proteins involved in blood ...

In contrast, hemophilia A is caused by the lack or insufficiency of clotting factor 8. [2] It is 3 to 4 times more common than hemophilia B and is also less severe. [1] ...

Hemophilia C is also distinct from hemophilia B, as it instead occurs due to alterations in the F11 gene and affects clotting factor XI. There are also less common conditions that affect other ...