Neose Technologies, Inc.

Amicus Therapeutics, Inc.

Eric Richard Sjoberg

Robert J Bayer

James C Paulson

Gary Lee

The present invention provides compounds and methods for the treatment of an individual having or at risk of having a condition associated with alpha-1-antitrypsin by using a pharmacological chaperone. In particular, such methods are useful for the treatment and/or prevention of lung disorders associated with alpha-1-antitrypsin deficiency as well as liver disorders associated with an excess of alpha-1-antitrypsin. Suitable pharmacological chaperones include peptides and low-molecular weight compounds. The present invention also provides an assay for determining whether a test compound modulates alpha-1-antitrypsin activity.

Compounds and method for treating or preventing disease conditions associated with alpha-1-antitrypsin

Pharmacological chaperone compounds and methods for the treatment of an individual having, or at risk of having, a disease condition associated with alpha-1-antitrypsin by using said compounds are disclosed. In particular, such methods are useful for the treatment or prevention of lung disorders associated with alpha-1-antitrypsin deficiency as well as liver disorders associated with an excess of alpha-1-antitrypsin. Suitable pharmacological chaperones include peptides and low-molecular weight compounds. Also provided is an assay for determining whether a test compound modulates alpha-1-antitrypsin activity.

Compounds and methods for treating or preventing disease conditions associated with &#x3b1;-1-antitrypsin

This invention provides methods for practical enzymatic conversion of GDP-mannose to GDP-fucose. These methods are useful for efficient synthesis of reactants used in the synthesis of fucosylated oligosaccharides.

Preparation of fucosylated oligosaccharides

This invention provides methods for practical in vitro sialylation of glycoproteins, including recombinantly produced glycoproteins. The methods are useful for large-scale modification of sialylation patterns.

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San Diego, CA, United States of America

Eric Richard Sjoberg