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For proposals focused on translational or clinical research to treat MPS I Scheie or MPSI HurlerScheie that have a strong likelihood of future federal funding or where the grant amount can be
matched. MPS I S/HS results from reduced enzymatic activity of alpha-L-iduronidase that leads to abnormal metabolic storage products and multi-organ pathologies. We are seeking
proposals for oral or parenteral drugs that will slow the progression of central nervous system (CNS) manifestations of this disease or new methods for measuring CNS disease progression,
including identification of novel disease-related structural or biochemical changes.