This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant. Sixteen patients will be enrolled over a 24 month period.
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Identify covariates that impact drug exposure
Timeframe: 2 years
Identify key differences pre- and post-HCT leading to variability in PK parameters
Timeframe: 2 years