Chaperone Therapy - Uses small molecule drugs that bind to the defective enzyme and stabilise it to increase enzyme activity and increase cellular function.

There are two brochures produced by Amicus Therapeutics that provide a better understanding of misfolded genes called "What's Missing?" (2007) and a better understanding of pharmcological chaperones and lysosomal storage disorders called "Pharmacological Chaperones" (2008). 
 

What’s Missing in Lysosomal Storage Diseases? (pdf - 6.5MB)

About Pharmacological Chaperones (pdf - 3.4MB)

In June 2009, Amicus announced the beginning of its Phase 3 study of AT1001, the company’s investigational drug for the treatment of Fabry disease.

For more information on the Fabry Phase 3 clinical trial, please visit

http://clinicaltrials.gov/ct2/show/NCT00925301