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Methods of Treating Sickle Cell Disease and Related Disorders Using Fumaric Acid Esters
专利权人:
发明人:
Vadivel Ganapathy
申请号:
US14105893
公开号:
US20140171504A1
申请日:
2013.12.13
申请国别(地区):
US
年份:
2014
代理人:
摘要:
Methods of using one or more fumaric acid esters or pharmacologically active salts, derivatives, analogues, or prodrugs thereof to increase expression of fetal hemoglobin (HbF) are disclosed. The methods typically include administering to a subject an effective amount of one or more fumaric acid esters optionally in combination or alternation with hydroxyurea to induce HbF expression in the subject in an effective amount to reduce one or more symptoms of a sickle cell disorder, a hemoglobinopathy, or a beta-thalassemia, or to compensate for a genetic mutation is the human beta-globin gene (HBB) or an expression control sequence thereof. Pharmaceutical dosage units and dosage regimes for use in the disclosed methods are also provided.
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