| abstract |
The present invention provides compositions and methods for effective treatment of a lysosomal acid lipase deficiency (LALD) disease, in particular, Wolman's disease and Cholesteryl Ester Storage Disease (CESD). Among other things, the present invention provides a method of treating a lysosomal acid lipase deficiency (LALD) disease, including administering to an individual suffering from or susceptible to the LALD disease a therapeutic effective amount of a lysosomal acid lipase periodically at an administration interval such that lipid level in liver, spleen and/or small intestine is reduced by at least 20% as compared to an untreated control. |