Methods for treating lysosomal acid lipase deficiency

摘要

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 developmental impairment or malnutrition in an individual suffering from a lysosomal acid lipase deficiency (LALD) disease, comprising administering to the individual a therapeutic effective amount of a lysosomal acid lipase.

主权项

1. A method of treating a lysosomal acid lipase deficiency (LALD) disease, comprising administering subcutaneously to an individual suffering from or susceptible to the LALD disease a therapeutically effective amount of a lysosomal acid lipase periodically at an administration interval, wherein a therapeutically effective amount of lysosomal acid lipase for the subcutaneous administration is about 5-fold more than a therapeutically effective amount of lysosomal acid lipase for intravenous administration.