title: Molecular approaches for improved clotting factors for hemophilia.
abstract: Hemophilia is caused by a functional deficiency of one of the coagulation proteins. Therapy for no other group of genetic diseases has seen the progress that has been made for hemophilia over the past 40 years, from a life expectancy in 1970 of ∼20 years for a boy born with severe hemophilia to essentially a normal life expectancy in 2013 with current prophylaxis therapy. However, these therapies are expensive and require IV infusions 3 to 4 times each week. These are exciting times for hemophilia because several new technologies that promise extended half-lives for factor products, with potential for improvements in quality of life for persons with hemophilia, are in late-phase clinical development.
authors: 1Degenerative Disease Research, Center for Neuroscience, Aging, and Stem Cell Research, Sanford Burnham Medical Research Institute, La Jolla, CA; and.
authors: Randal J
authors: RJ
authors: Kaufman
authors: Jerry S
authors: JS
authors: Powell
mesh_terms: D001779:Blood Coagulation Factors / Q000627:therapeutic use*; D005260:Female; D006467:Hemophilia A / Q000188:drug therapy* / Q000266:history / Q000401:mortality; D049673:History, 20th Century; D049674:History, 21st Century; D006801:Humans; D008017:Life Expectancy; D008297:Male; D011788:Quality of Life
chemical_list: D001779:Blood Coagulation Factors