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Title:
イズロン酸−2−スルファターゼのCNS送達のための方法および組成物
Document Type and Number:
Japanese Patent JP6938456
Kind Code:
B2
Abstract:
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.

Inventors:
Gaochon Chu
Chris lowe
Zara Sharok
James christian
Rick furner
Zinpan
Teresa rear light
Pellicles Carias
Application Number:
JP2018227369A
Publication Date:
September 22, 2021
Filing Date:
December 04, 2018
Export Citation:
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Assignee:
Shire Human Genetic Therapy Inc.
International Classes:
A61K38/46; A61K9/08; A61K9/19; A61K47/04; A61K47/26; A61M5/178; A61P25/00; A61P43/00
Domestic Patent References:
JP2007504166A
Other References:
Eur. J. Pediatr.,2008年,Vol.167,pp.267-277
Attorney, Agent or Firm:
Hidesaku Yamamoto
Natsuki Morishita
Takatoshi Iida
Daisuke Ishikawa
Kensaku Yamamoto