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.
申请公布号
AU2011270668(B8)
申请公布日期
2017.02.09
申请号
AU20110270668
申请日期
2011.06.25
申请人
Shire Human Genetic Therapies, Inc.
发明人
Calias, Pericles;Pan, Jing;Powell, Jan;Charnas, Lawrence;McCauley, Thomas;Shahrokh, Zahra;Wright, Teresa Leah;Pfeifer, Richard