Future Developments for Gaucher Disease


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Amicus Therapeutics, a biopharmaceutical company based in New Jersey USA, is developing a series of drugs called small molecule pharmacological chaperones to treat human genetic diseases and is planning to initiate clinical studies of a small molecule chaperone called AT2101 for Gaucher disease in the first six months of 2006.

The idea behind this method of treatment is to enable the patient's own enzyme to work more effectively. Enzyme replacement therapy (Cerezyme) infuses more enzyme into a patient whilst substrate reduction therapy (Zavesca) reduces the amount of fatty material (glucocerebroside) so that the patient has enough enzyme to dispose of it.

Therefore a small molecule chaperone is a potential third method of treatment which hopefully will provide a patient with enough enzyme to prevent the symptoms of Gaucher disease.


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Source: Gauchers News December 2005.
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