Improvements in MPS-HAQ domain scores vs. baseline at 1 and 2 years and a non-significant decrease in the caregiver-assistance domain at 2 years. The MOR-005 intent-to-treat population (ITT N = 169, including 158 with 2 years follow-up) showed sustained significant reductions (representing improvements) in mobility and self-care domain least square (LS) mean scores vs. Mean baseline MPS-HAQ domain scores showed impairments in mobility, self-care, and independence.