Amicus Therapeutics, Inc.

P2d, Inc.

The United States of America, As Represented by the Secretary, Department of Health and Human Services

Giordano, Tony

Brossi, Arnold

Xiaoxiang Zhu

Philip J Rybczynski

Nigel H Greig

Arnold Brossi

Harold W Holloway

William Douglas Figg

Kamlesh A Sheth

Qian-Sheng Yu

Tony Giordano

Robert E Boyd

Gary Lee

Benjamin Mugrage

Jian-Qiang Fan

Kamlesh Sheth

Dan Fuerst

Alexander Tretyakov

Benjamin Mugrace

William D Figg

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

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

Thalidomide analogs that modulate tumor necrosis factor alpha (TNF&#x3b1;) activity and angiogenesis are disclosed. In particularly disclosed embodiments, the thalidomide analogs are isosteric sulfur-containing analogs. Also disclosed are methods of treating a subject with the analogs.

Thalidomide analogs

Thalidomide analogs that modulate tumor necrosis factor alpha (TNF-&#x3b1;) activity and angiogenesis are disclosed. In particularly disclosed embodiments, the thalidomide analogs are isosteric sulfur-containing analogs. Also disclosed are methods of treating a subject with the analogs.

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.

Hydroxy piperidine derivatives to treat gaucher disease

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North Brunswick, NJ, United States of America

Xiaoxiang Zhu