METHODS OF USING INHIBITORS OF PIKFYVE FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS AND NEURODEGENERATIVE DISEASES

摘要

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.