The United States of America, As Represented by the Secretary, Department of Health and Human Services

Emory University

The United States of America, As Represented by National Institute of Health

Marjan Huizing

William A Gahl

Irini Manoli

Enriko Klootwijk

Rong Jiang

Nuria Carrillo

Xueli Li

May C Malicdan

Nuria Carrillo-Carrasco

Miao He

The invention relates to compositions and methods for treating kidney and muscle dysfunction that involves use of therapeutic amounts of N-acetyl mannosamine.

N-acetyl mannosammine as a therapeutic agent

Methods are disclosed for treating a subject with a vascular or cardiac disorder associated with oxidative stress. Methods are disclosed for treating a subject with GNE myopathy that has impaired cardiac function. These methods include administering to the subject a therapeutically effective amount of a acid, or one or more sialylated compounds, mannosamine, N-acetyl mannosamine or a derivative thereof. In other embodiments, methods are disclosed for detecting a disorder associated with oxidative stress.

Sialylation-increasing therapies for diseases associated with oxidative stress

Methods are disclosed for diagnosing a hyposialylation disorder. Methods are also disclosed for determining the effectiveness of a therapeutic agent for treatment of a hyposialylation disorder in a subject. These methods include measuring an amount of monosialylated Thomsen-Friedenreich (ST) antigen and measuring an amount of non-sialylated Thomsen-Friedenreich antigen (T) in a biological sample, such as a serum or plasma sample from the subject and determining the ratio of T to ST. A ratio of T to monosialylated ST of about 0.06 or higher diagnoses the hyposialylation disorder or indicates that the therapeutic agent is not effective for the treatment of the hyposialylation disorder. In other embodiments, a ratio of T to ST less than about 0.06 indicates that the therapeutic agent is effective for the treatment of the hyposialylation disorder, or the subject does not have the hyposialylation disorder. In additional embodiments, these methods can be used to determine the lowest effective dosage of the therapeutic agent of use to treat the subject.

Hyposialylation disorders

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N-acetyl mannosamine as a therapeutic agent

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Santa Cruz, CA, United States of America

Marjan Huizing