Pompe disease is a rare genetic condition that leads to a buildup of the complex sugar glycogen, causing muscular impairment. The condition may also lead to heart failure in young patients. The U.S. Food and Drug Administration (FDA) in October had extended the review of the drug for the second time, blaming the delay on pandemic-related travel restrictions that kept it from conducting a required inspection of the manufacturing site. Amicus expects a recommendation on miglustat in the second quarter of 2023. (This story has been corrected to say 2023, not 2022, in the last paragraph)
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