Methods for treating farber disease

A method of treating a human subject with Farber disease, comprising administering to the human subject a therapeutically effective dose of recombinant human acid ceramidase (rhAC) wherein the therapeutically effective dose may be determined based on the pharmacokinetic profile obtained in juvenile, healthy CD-1 mice receiving a single, maximum effective dose of 10 mg/kg of rhAC intraperitoneally, wherein the rhAC elicits in the healthy CD-1 mice one or more of:

• Tmax of about 0.25 to 1.0 hours, Cmax of about 1.23 to about 2.17 μg/mL, or area under the curve (AUC) of about 1 hr*μg/mL to about 2 hr*μg/mL.

A method of treating a human subject with Farber disease, comprising administering to the subject a therapeutically effective dose of rhAC wherein the therapeutically effective dose may be determined based on the pharmacokinetic profile, obtained in juvenile, healthy CD-1 mice receiving a single, maximum effective dose (“MED”) of 10 mg/kg dose of rhAC intraperitoneally, wherein the rhAC elicits in the healthy CD-1 mouse a Cmax, AUC0-last or AUCtissue/AUCserum in accordance with the values set forth in FIG. 7.