N-acetyl mannosamine as a therapeutic agent
AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Services
InventorsMarjan Huizing, William A. Gahl, Irini Manoli, Enriko Klootwijk
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Abstract
The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.
Claims20
A method of treating a kidney disease or condition in a mammal in need thereof comprising administering orally an effective amount of a sialic acid precursor, N-acetyl mannosamine or a derivative thereof, to the mammal to thereby treat a kidney disease or condition in the mammal, wherein the derivative consists of Formula I;
- wherein:
- R1, R3, R4, or R5 is hydrogen, lower alkanoyl, carboxylate or lower alkyl;
- and R2 is lower alkyl, lower alkanoylalkyl, or lower alkyl alkanoyloxy, wherein the kidney disease or condition is selected from podocytopathy, minimal change nephrosis, focal and segmental glomerulosclerosis, membranous glomerulonephritis, glomerular basement membrane disease, nephrotic syndrome or other kidney disease or condition with hyposialylation.
A method of treating distal myopathy with rimmed vacuoles (DMRV, Nonaka myopathy) or muscular dystrophy hereditary inclusion body myopathy (HIBM) in a mammal in need thereof comprising administering an effective amount of N-acetyl mannosamine to the mammal to thereby treat the DMRV or HIBM in the mammal.
Dependencies are inferred from the claim text.