Other

Amicus Therapeutics, Inc.

Errico, Joseph P.

Benjamin Mugrage

Joseph P Errico

Jane Ong

John Allocco

Pam Wines

Matthew Sills

Ignatius Turchi

Kamlesh A Sheth

Philip J Rybczynski

Robert E Boyd

David Palling

Gary Lee

Margarita Bastos

Xiaoxiang Zhu

The present invention provides a method for increasing the activity of a mutant or wild-type &#x3b1;-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 &#x3b1;-glucosidase gene or enzyme is also provided.

Method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives

Provided is a method of treating a proliferative disease, condition, or disorder in a subject by administering a combination of an inhibitor of p53 and MDM2 binding and an EGFR inhibitor. Various embodiments of the disclosed methods provide a synergistic anti-proliferative or anti-apoptotic effect compared to administration of one agent alone.

Combination therapy with MDM2 and EFGR inhibitors

Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives

Provided herein are compounds having anti-proliferative effect. Also provided are compounds that can modulate the activity of multi-domain proteins comprising a dimerization arm and interdomain tether, such as EGFR, where an untethered, extended conformation is the active state and a tethered conformation is the inactive state, resulting in an autoinhibited configuration. Also provided are methods and pharmacophores for identifying such compounds. Other aspects provide methods or therapeutic treatment for proliferative diseases, disorders, or conditions, such as those associated with EGFR.

Methods of targeted drug development

Methods and compositions of targeted drug development

A novel tartaric acid salt of Isofagomine (Isofagomine tartrate) that can be used for the treatment of Gaucher disease is provided. The invention also provides a crystalline form of isofagomine tartrate, method for preparing the salt, a pharmaceutical composition containing the salt, and a method of treating Gaucher disease.

Tartrate salt of isofagomine and methods of use

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Cranbury, NJ, United States of America

Benjamin Mugrage