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Study: Pompe More Common Than Previously Estimated

The number of people with Pompe disease varies according to ethnic group and may be higher among all ethnicities than previously estimated, a study suggests. According to the investigator, the study stresses the importance of analyzing genetic disorders in the general population to improve predictive and preventive medicine. “With this…

FDA Filing of AT-GAA for Late-onset Pompe Expected by Mid-Year

Amicus Therapeutics anticipates completing a rolling application by mid-year to seek approval of its investigational therapy AT-GAA for late-onset Pompe disease in the U.S. The announcement follows a pre-filing meeting with the U.S. Food and Drug Administration (FDA). The rolling biologics license application (BLA), initiated in…