Molecular approaches for improved clotting factors for hemophilia.

Randal J. Kaufman, Jerry S Powell

Research output: Contribution to journalArticle

28 Citations (Scopus)

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 languageEnglish (US)
Pages (from-to)3568-3574
Number of pages7
JournalBlood
Volume122
Issue number22
StatePublished - Nov 21 2013

Fingerprint

Blood Coagulation Factors
Hemophilia A
Coagulation
Life Expectancy
Proteins
Inborn Genetic Diseases
Therapeutics
Quality of Life
Technology

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology
  • Medicine(all)

Cite this

Molecular approaches for improved clotting factors for hemophilia. / Kaufman, Randal J.; Powell, Jerry S.

In: Blood, Vol. 122, No. 22, 21.11.2013, p. 3568-3574.

Research output: Contribution to journalArticle

Kaufman, RJ & Powell, JS 2013, 'Molecular approaches for improved clotting factors for hemophilia.', Blood, vol. 122, no. 22, pp. 3568-3574.
Kaufman, Randal J. ; Powell, Jerry S. / Molecular approaches for improved clotting factors for hemophilia. In: Blood. 2013 ; Vol. 122, No. 22. pp. 3568-3574.
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