Secondary Outcome(s)
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Liver Volumes (Body Weight Adjusted) in Part 1
[Time Frame: Baseline to Week 5]
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Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 2
[Time Frame: Baseline to Week 12]
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Heparan Sulfate Concentrations in Serum in Part 2
[Time Frame: Baseline to Week 13]
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Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 1
[Time Frame: Baseline to Week 4]
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Dermatan Sulfate Concentrations in Serum in Part 1
[Time Frame: Baseline to Week 5]
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Liver Volumes (Body Weight Adjusted) in Part 2
[Time Frame: Baseline to Week 13]
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Spleen Volumes (Body Weight Adjusted) in Part 2
[Time Frame: Baseline to Week 13]
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Heparan Sulfate Concentrations in Urine in Part 1
[Time Frame: Baseline to Week 5]
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Pharmacokinetics of Each Dose in Part 1 (Cmax)
[Time Frame: Week 1 to Week 4]
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Dermatan Sulfate Concentrations in Cerebrospinal Fluid in Part 2
[Time Frame: Baseline to Week 12]
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Heparan Sulfate Concentrations in Serum in Part 1
[Time Frame: Baseline to Week 5]
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Heparan Sulfate Concentrations in Urine in Part 2
[Time Frame: Baseline to Week 13]
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Pharmacokinetics of Each Dose in Part 2 (Cmax)
[Time Frame: Week 1 to Week 12]
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Dermatan Sulfate Concentrations in Urine in Part 2
[Time Frame: Baseline to Week 13]
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Pharmacokinetics of Each Dose in Part 2 (AUC0-t)
[Time Frame: Week 1 to Week 12]
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Spleen Volumes (Body Weight Adjusted) in Part 1
[Time Frame: Baseline to Week 5]
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Heparan Sulfate Concentrations in Cerebrospinal Fluid in Part 1
[Time Frame: Baseline to Week 4]
|
Dermatan Sulfate Concentrations in Serum in Part 2
[Time Frame: Baseline to Week 13]
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Dermatan Sulfate Concentrations in Urine in Part 1
[Time Frame: Baseline to Week 5]
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Pharmacokinetics of Each Dose in Part 1 (AUC0-t)
[Time Frame: Week 1 to Week 4]
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