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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…
Enhancing of homing and/or engraftment of cord blood mononuclear cells in the central nervous system
Value Proposition Leukodystrophies are inherited disorders resulting in decreased motor function, muscle rigidity, degeneration of sight and hearing, and are ultimately fatal. There is a great lack of treatment, although cord blood mononuclear (CBM) and…