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

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 effec...

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Bibliographic Details
Main Authors BHAGWAT, Shripad, POST, Leonard, CRAWFORD, Brett E
Format Patent
LanguageEnglish
French
Published 09.03.2017
Subjects
CHEMISTRY
HETEROCYCLIC COMPOUNDS
METALLURGY
ORGANIC CHEMISTRY
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Summary: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. Cette invention concerne des méthodes de traitement des troubles du stockage lysosomal (par exemple les MPS) et/ou des maladies neurodégénératives (par exemple la maladie d'Alzheimer), qui consistent à administrer à un sujet (par exemple un patient humain) ayant besoin d'un tel traitement (par exemple, identifié comme nécessitant un tel traitement) une quantité efficace d'un ou de plusieurs inhibiteurs de PIKfyve, ou d'un sel de qualité pharmaceutique de ceux-ci.
Bibliography:Application Number: WO2016US50162