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14-1530B A Novel Investigational Treatment in Preclinical Development for Galactosemia

Shoshana Shendelman, PhD

Marina Brodsky, PhD

Clinical complications in galactosemia have been associated with accumulation of galactose metabolites. Preventing or reducing the formation of potentially toxic metabolites may reduce the risk of complications in galactosemia patients.   Applied Therapeutics is developing a novel investigational compound to block formation of certain galactose metabolites, which may be responsible for galactosemia complications.  Please join us to learn more about our science, treatment strategy, preclinical results and future clinical development plans.