Compositions and methods for treating muscular dystrophy and other disorders
AssigneeThe Charlotte-Mecklenburg Hospital Authority
InventorQi Long Lu
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Abstract
The present invention provides compositions and methods of their use in treating muscular dystrophy and other disorders.
Claims2
A method of treating a disorder associated with a mutation or loss of function in a fukutin related protein (FKRP) gene in a subject, comprising administering a therapeutically effective amount of a ribitol to a subject in need thereof daily for at least 30 days, thereby treating the disorder associated with a mutation or loss of function in a fukutin related protein (FKRP) gene in a subject, wherein the disorder associated with a mutation or loss of function in the FKRP gene is selected from the group consisting of limb-girdle muscular dystrophy (LGMD), Walker-Warburg syndrome (WWS), muscle-eye-brain disease (MEB), congenital muscular dystrophy (CMD), and any combination thereof.
Dependencies are inferred from the claim text.