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Showing 4 matches for the following: Inventor: "Kishnani, Priya" ×
Muscle mass influences upon response to therapy
Duke University is seeking a company interested in commercializing a drug treatment method for enhancing lysosomal storage disease therapy by modulating cell surface receptor expression. Lysosmal storage diseases (LSDs), including Pompe disease, Gaucher disease, Farby…
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…
Test of Verbal Expression and Reasoning (TOVER)
The TOVER is non-standardized assessment of expressive language in context and verbal reasoning. The 64 item test was specifically designed to assess language function in children and adults with Down syndrome across a broad range…
Methods of treatment in diseases treated with therapeutic proteins
The present invention relates, in general, to a method of treating patients undergoing enzyme replacement therapy (ERT) or other therapy involving the administration of a proteinaceous therapeutic agent as well gene replacement therapy with non-viral…