Novel valproic acid derivatives with hemoglobin F inducing activity

American Journal of Hematology
G RönndahlO Witt

Abstract

Pharmacological induction of hemoglobin F expression may be a promising approach for the treatment of beta-thalassemia and sickle cell disease. Valproic acid, a drug frequently used for the treatment of seizure disorders, has been shown to enhance fetal hemoglobin synthesis in erythroid cells. However, this effect is only modest and requires relative high concentrations. Therefore, the drug appears not to be applicable for the treatment of beta-globin chain disorders. Here, we describe the identification of novel valproic acid derivatives with potent hemoglobin F inducing activities at concentrations that presumably can be obtained in vivo.

References

Sep 15, 1998·Human Molecular Genetics·N F Olivieri, D J Weatherall
Sep 11, 2002·American Journal of Hematology·O WittA Pekrun
Apr 12, 2003·Pediatric Hematology and Oncology·M KieslichP Hernáiz Driever

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Citations

Sep 29, 2009·Expert Opinion on Investigational Drugs·Sandy D Kotiah, Samir K Ballas
Nov 14, 2008·Expert Opinion on Pharmacotherapy·Winfred C Wang

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