The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Nov. 10, 2015
Filed:
May. 17, 2006
Benjamin Mugrage, Cranbury, NJ (US);
Gary Lee, Cranbury, NJ (US);
Xiaoxiang Zhu, Cranbury, NJ (US);
Robert Boyd, Cranbury, NJ (US);
Kamlesh Sheth, Cranbury, NJ (US);
Benjamin Mugrage, Cranbury, NJ (US);
Gary Lee, Cranbury, NJ (US);
Xiaoxiang Zhu, Cranbury, NJ (US);
Robert Boyd, Cranbury, NJ (US);
Kamlesh Sheth, Cranbury, NJ (US);
Amicus Therapeutics, Inc., Cranbury, NJ (US);
Abstract
The present invention provides a method for increasing the activity of a mutant or wild-type α-glucosidase enzyme in vitro and in vivo by contacting the enzyme with a specific pharmacological chaperone which is a derivative of 1-deoxynojirimycin. The invention also provides a method for the treatment of Pompe disease by administration of chaperone small molecule compound which is a derivative of 1-deoxynojirimycin. The 1-deoxynojirimycin derivative is substituted at the N or C1 position. Combination therapy with replacement α-glucosidase gene or enzyme is also provided.