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.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 30-36 |
| Number of pages | 7 |
| Journal | Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program |
| Volume | 2013 |
| DOIs | |
| State | Published - 2013 |
| Externally published | Yes |
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ASJC Scopus subject areas
- Medicine(all)
Cite this
Molecular approaches for improved clotting factors for hemophilia. / Kaufman, Randal J.; Powell, Jerry S.
In: Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program, Vol. 2013, 2013, p. 30-36.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Molecular approaches for improved clotting factors for hemophilia
AU - Kaufman, Randal J.
AU - Powell, Jerry S
PY - 2013
Y1 - 2013
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=84907051813&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84907051813&partnerID=8YFLogxK
U2 - 10.1182/asheducation-2013.1.30
DO - 10.1182/asheducation-2013.1.30
M3 - Article
C2 - 24319159
AN - SCOPUS:84907051813
VL - 2013
SP - 30
EP - 36
JO - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
JF - Hematology / the Education Program of the American Society of Hematology. American Society of Hematology. Education Program
SN - 1520-4391
ER -