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Percentage CFBL in a serum CK and b urine Hex4 in ambulatory patients over 48 months. Pooled data from cohorts 1, and 4 for ERT-experienced patients. aLower normal CK limit = 26 IU/L, upper normal CK limit = 192 IU/L. CFBL change from baseline, CK serum creatine kinase, ERT enzyme replacement therapy, Hex4 urine glucose tetrasaccharide, SD standard deviation, SE standard error
