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1. (WO2017040971) METHODS OF USING INHIBITORS OF PIKFYVE FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS AND NEURODEGENERATIVE DISEASES

Pub. No.:    WO/2017/040971    International Application No.:    PCT/US2016/050162
Publication Date: Fri Mar 10 00:59:59 CET 2017 International Filing Date: Sat Sep 03 01:59:59 CEST 2016
IPC: C07D 239/00
C07D 239/48
C07D 413/04
Applicants: BIOMARIN PHARMACEUTICAL INC.
Inventors: BHAGWAT, Shripad
CRAWFORD, Brett E.
POST, Leonard
Title: METHODS OF USING INHIBITORS OF PIKFYVE FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS AND NEURODEGENERATIVE DISEASES
Abstract:
This disclosure features methods for treating lysosomal storage disorders (e.g. MPS) and/or neurodegenerative diseases (e.g., Alzheimers disease), which include administering to a subject (e.g., a human patient) in need of such treatment (e.g., identified as being in need of such treatment) an effective amount of one or more PIKfyve inhibitors, or a pharmaceutically acceptable salt thereof.