Methods for Treating Lysosomal Acid Lipase Deficiency

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...

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Main Authors MARTINI PAOLO, CHARNAS LAWRENCE, MEIYAPPAN MUTHURAMAN, CONCINO MICHAEL, HEARTLEIN MICHAEL, DU HONG, GRABOWSKI GREGORY, ROMASHKO ALLA, PESCATORE BRIAN
Format Patent
LanguageEnglish
Published 12.12.2013
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Summary: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.
Bibliography:Application Number: US201313967446