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US 10,751,387 B2Granted
Pharmacological therapy for neuronopathic Gaucher disease
AssigneeChildren's Hospital Medical Center
InventorsYing Sun, Benjamin Liou
Life of this patent
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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.
Claims17
A method of treating an individual having neuronopathic Gaucher disease (nGD), comprising
- a) administering an effective amount of a ryanodine receptor inhibitor selected from dantrolene, JTV-519, flecainide-d3, flecainide, 4-(2-Aminopropyl)-3,5-dichloro-N,N-dimethylaniline (FLA 365), DHBP (1,1′-diheptyl-4,4′-bipyridium), ruthenium red (R2751), or a combination thereof, to said individual, wherein said administration is in an amount sufficient to reduce brain inflammation in said individual;
- and
- b) monitoring gait impairment in said individual during said treatment.
Dependencies are inferred from the claim text.