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AssigneeAugusta University Research Institute, Inc.
InventorsVadivel Ganapathy, Pamela M. Martin
Estimating the term…
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.
A method of treating a sickle cell disorder or complication thereof or treating sickle cell retinopathy in subject comprising administering to the subject an effective amount of a fumaric acid ester, or pharmacologically active salt, derivative, analogue, or prodrug thereof to increase fetal hemoglobin (HbF) expression in the subject.
A method of treating sickle cell disease in a subject in need thereof comprising administering to the subject in combination or alternation effective amounts of a fumaric acid ester and hydroxyurea (HU) to induce expression of HbF in erythroid or retinal cells of the subject.
(canceled)
A pharmaceutical composition comprising an effective amount of a fumaric acid ester in combination with HU to induce expression of HbF in human erythroid or retinal cells.
Dependencies are inferred from the claim text.