US Patent 7741340 Hydroxy piperidine derivatives to treat gaucher disease
Patent 7741340 was granted and assigned to Amicus Therapeutics on June, 2010 by the United States Patent and Trademark Office.
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The present invention provides novel hydroxy piperidine (HP) derivatives having (i) a positive charge at the position corresponding to the anomeric position of a pyranose ring; (ii) a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and (iii) a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel HP derivative as “active-site specific chaperones” for the mutant glucocerebrosidase associated with the disease.
US Patent 07741340 Hydroxy piperidine derivatives to treat gaucher disease US Patent 7741340 Hydroxy piperidine derivatives to treat gaucher disease
US Patent 07741340 Hydroxy piperidine derivatives to treat gaucher disease
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