Article: Amicus Therapeutics Files Investigational New Drug Application for AT2101 for Gaucher Disease.

Phase I Clinical Trials Expected to Commence in June

CRANBURY, N.J., April 27 /PRNewswire/ -- Amicus Therapeutics, a biopharmaceutical company developing small molecule, orally-administered pharmacological chaperones for the treatment of human genetic diseases, today announced that it has submitted an investigational new drug application (IND) to the U.S. Food and Drug Administration (FDA) for AT2101 for the treatment of Gaucher disease. Pending FDA review of the IND, the company plans to begin Phase I clinical trials in June.

Gaucher disease is a lysosomal storage disorder that affects approximately 10,000 people in the developed world. Symptoms can ...

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