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Fety and effectiveness of galafold have not been established in pediatric patients Early initiation of ert in childhood or young adulthood was associated with better renal and cardiac outcomes than treatment initiation at a later age. Migalastat (galafold™), a pharmacological chaperone, stabilizes and facilitates trafficking of.

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Gb3 accumulation occurs in virtually all cardiac cells (cardiomyocytes, conduction system cells. Enzyme replacement therapy (ert) with agalsidase alfa or agalsidase beta stabilized renal function and cardiac structure in patients with fabry disease