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For the second approved Fabry disease therapy, the chaperone therapy, it was shown that administration of chaperones galactose and 1-deoxygalactonojirimycin (DGJ, migalastat) can help to improve the enzyme activity in cultured fibroblasts by stabilizing the mutant protein so it can be transported to the lysosome and execute its catalytic function [41]

The approach described in this section would identify PDE records as potentially 340B-eligible based on two criteria: (1) the prescriber with the NPI listed on the PDE record provides care at a covered entity, and (2) the pharmacy NPI on the PDE record is a contract pharmacy for that same covered entity

Mammary stem cells in domestic animals: the role of ROS