Cincinnati Children's Hospital Medical Center

Benjamin Liou

Gregory A Grabowski

Ying Sun

The instant disclosure relates to methods and compositions for the treatment of Gaucher disease, particularly type II and III neuronopathic Gaucher disease (nGD). The methods include the step of administering to an individual in need thereof an effective amount of a ryanodine receptor inhibitor or a pharmaceutically acceptable salt thereof.

Pharmacological therapy for neuronopathic Gaucher disease

The present disclosure relates to methods of treating diseases states such as lysosomal storage diseases and/or neurodegenerative diseases. Also disclosed are one or more compositions that may be useful for one or more of the disclosed methods, including compositions that may comprise acid &#x3b2;-glucosidase (GCase) protein comprising one or more mutations, peptides of acid &#x3b2;-glucosidase, and DNA vectors and cell lines related to acid &#x3b2;-glucosidase peptides or proteins.

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Cincinnati, OH, United States of America

Benjamin Liou