Washington University

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

Fumihiko Urano

Simin Lu

David Joseph Maloney

Ajit Jadhav

Shyh-Ming Yang

Ying Chen

Yeawon Kim

Amy Clark

Sun-Ji Park

Brandon Harvey

Mark Henderson

Jana Mahadevan

The present invention generally relates to compositions and methods for treating or preventing an endoplasmic reticulum stress disorder in subjects, including compositions and methods for treating or preventing Wolfram syndrome. The present invention also relates to methods for treating symptoms of neurodegenerative diseases.

Treatment for Wolfram syndrome and other endoplasmic reticulum stress disorders

Among the various aspects of the present disclosure is the provision of methods and compositions to treat or prevent an endoplasmic reticulum (ER) stress-mediated kidney disease in a subject, the method comprising administering to the subject a pharmaceutical composition comprising a therapeutically effective amount of an ER calcium channel stabilizing agent or an ER stress modulating agent (e.g., mesencephalic astrocyte-derived neurotrophic factor (MANF), compound K201).

Compositions and methods for treating and preventing endoplasmic reticulum (ER) stress-mediated kidney diseases

The present invention generally relates to a method of treating or preventing an endoplasmic reticulum stress disorder in subjects, such as a method of treating or preventing Wolfram syndrome.

Treatment for wolfram syndrome and other endoplasmic reticulum stress disorders

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St. Louis, MO, United States of America

Fumihiko Urano