Methods and compositions for the treatment of steatosis-associated disorders
AssigneesDuke University, National University of Singapore
InventorsDwight D. Koeberl, Paul M. Yen, Benjamin L. Farah
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Abstract
The present disclosure is directed to methods of treating a steatosis- associated disorder by administering a therapeutic agent selected from a lysosomal enzyme, an autophagy-inducing agent, or a combination thereof. Steatosis-associated disorders discussed herein include GSD Ia, GSD Ib, GSD Ic, NAFLD, and NASH. Other embodiments are directed to methods of reversing steatosis, modulating autophagy, inducing autophagy, and reversing glycogen storage.
Claims17
A method of treating a subject, the method comprising:
- administering to a subject having Glycogen Storage Disease Type I (GSD I) a therapeutically effective amount of fenofibrate;
- and
- administering to the subject a therapeutically effective amount of acid alpha-glucosidase or recombinant acid alpha-glucosidase,
- wherein, following the administering steps, one or more GSD I symptoms are improved and/or one or more long-term GSD I complications are prevented and/or delayed.
A method of reversing steatosis in a subject, the method comprising:
- administering to a subject having a steatosis-associated disorder a therapeutically effective amount of fenofibrate, wherein the steatosis-associated disorder is Glycogen Storage Disease Type I (GSD I), non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), or any combination thereof;
- and
- administering to the subject a therapeutically effect amount of acid alpha-glucosidase or recombinant acid alpha-glucosidase,
- wherein, following the administering steps, the subject is treated by a reduction or prevention of one or more of hepatosteatosis, abdominal discomfort, elevated liver enzyme levels, fatigue, malaise, hepatomegaly, hyperlipidemia, hypoglycemia, hypertension, iron-resistant anemia, kidney stones, growth delay, lactic academia, nephropathy, hepatic/renal glycogenosis, pancreatitis, hepatic adenomata, hepatocellular carcinoma, osteopenia/osteoporosis, platelet dysfunction, spider angiomata, ascites, splenomegaly, hard liver border, palmar erythema, or asterixis.
Dependencies are inferred from the claim text.