Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Synageva Biopharma Corporation

Jan Powell

Pericles Calias

Lawrence Charnas

Zahra Shahrokh

Jing Pan

Thomas Mccauley

Teresa Leah Wright

Richard Pfeifer

Michael W Heartlein

Michael F Concino

Paolo Martini

Muthuraman Meiyappan

Alla Romashko

Brian Felice

Brian Pescatore

Nancy Savioli

The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.

CNS delivery of therapeutic agents

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.

Methods for treating lysosomal acid lipase deficiency

Growing community of inventors

Concord, MA, United States of America

Jan Powell