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1. (WO2018183940) METHODS AND COMPOSITIONS FOR TREATING NIEMANN-PICK DISEASE

Pub. No.:    WO/2018/183940    International Application No.:    PCT/US2018/025539
Publication Date: Fri Oct 05 01:59:59 CEST 2018 International Filing Date: Sat Mar 31 01:59:59 CEST 2018
IPC: A61K 31/192
A61K 9/00
Applicants: NEUROVIA, INC.
Inventors: GRASS, IV, George Mitchell
Title: METHODS AND COMPOSITIONS FOR TREATING NIEMANN-PICK DISEASE
Abstract:
Disclosed are methods for treating Niemann-Pick disease (e.g., Niemann-Pick disease type C (e.g., type C1)) by administering an effective amount of sobetirome or a sobetirome prodrug to a patient having the Niemann-Pick disease (e.g., Niemann-Pick disease type C (e.g., type C1)). Also disclosed are compositions containing sobetirome or a sobetirome prodrug for use in treating Niemann-Pick disease (e.g., Niemann-Pick disease type C (e.g., type C1)).