Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome.
Elsa G ShapiroChester B WhitleyJulie B EisengartPublished in: Orphanet journal of rare diseases (2018)
This report enables clarification of functioning at levels that extend beneath the floor of 50 in previous work. At the dawn of newborn screening and amidst a proliferation of new therapies for MPS I, these data can provide crucial benchmark information for developing treatments, particularly for areas of the world where transplant may not be available.