Discovery of brain MRI signatures in infants with severe form of MPS I in the pre-HSCT and post-HSCT stage

PI: Igor Nestrasil, MD

Institution: University of Minnesota

MPS Statistics

Institutions Awarded


Countries Awarded


# of Awards


Total Amount


Award Period: 2016-2017

Award Total: $165,000


Award Period: 2017-2018

Award Total $165,543

The overall goal of the project is to measure the brain magnetic resonance imaging (MRI) abnormalities in infants and very young children with the severe form of mucopolysaccharidosis type I (MPS IH) before they undergo hematopoietic stem cell transplantation (HSCT). These critical MRI data is currently not available. We established MRI outcomes for detection of abnormalities examining brain volumes, myelin content, and white matter microstructural integrity in the brain of MPSIH infants and very young children and compared them to age-matched healthy controls using the identical imaging protocol and analysis.