Novel pharmacological therapy for neuronopathic gaucher disease
AssigneeChildren's Hospital Medical Center
InventorsYing Sun, Benjamin Liou
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Abstract
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.
Claims9
A method of treating a condition associated with neuronopathic Gaucher disease (nGD), in an individual in need thereof, comprising administering an effective amount of dantrolene or pharmaceutically acceptable salt thereof to said individual wherein said condition is selected from one or more of brain inflammation, neuron loss, and neurodegeneration.
4-13. (canceled)
A method for a neurological sign in an individual having nGD, comprising administering dantrolene, or pharmaceutically salt thereof, to said individual.
Dependencies are inferred from the claim text.