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Novel Method for Treating Steatosis-Associated Diseases
Unmet Need Steatosis-associated diseases are marked by the infiltration of the liver with fatty acids. Liver steatosis occurs in nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH), as well as rare diseases such as…
Method for immune tolerance induction in patients receiving enzyme replacement therapy for a variety of metabolic disorders
Unmet Need Genetically based metabolic disorders affect 1 in 2500 births globally and many of these disorders result in an enzyme deficiency. In most cases, a single enzyme is not produced by the body at…
A method of screening and monitoring glycogen storage diseases with a single biomarker
Unmet Need Glycogen storage disease type I (GSD I) is an autosomal recessive inherited disorder of carbohydrate metabolism associated with life threatening fasting hypoglycemia, and significant morbidity and mortality. Currently, the standard of care therapy…