ABSTRACT
Safety and Tolerance .............................................................................................342 Effects on Spleen and Liver — Volume and Functions .......................................343 Hematological Response .......................................................................................345 Bone Changes on ERT ..........................................................................................346 Other Clinical Effects ............................................................................................347 Biomarkers and Other Laboratory Findings .........................................................348 On Dose and Individualization..............................................................................349 Indications for ERT and Timing of Its Beginning................................................349 Maintenance Regimens and the Effects of Withdrawal of Therapy.....................351 New Enzymatic Preparations ................................................................................351 Conclusions............................................................................................................352 References..............................................................................................................352
