Fabry disease

Treatment

Why do I have to take Galafold every other day?

Galafold works by helping to stabilize the faulty enzyme in Fabry disease and guiding it into the lysosomes to help break down accumulated lipid waste. Therefore, it is called a chaperone therapy. Once the enzyme has entered the lysosome, the chaperone will detach from the enzyme so it can break down the waste. The every-other-day dosing allows time for the protein to detach and let the enzyme complete its work. This is a case where more is not necessarily better.

Galafold: An oral treatment option for certain people with Fabry disease. (n.d.). Retrieved from http://www.galafold.com/.

Fabry disease revisited: Management and treatment recommendations for adult patients. Ortiz A, Germain DP, Desnick RJ, Politei J, Mauer M, Burlina A, Eng C, Hopkin RJ, Laney D, Linhart A, Waldek S, Wallace E, Weidemann F, Wilcox WR. Mol Genet Metab. 2018 Apr;123(4):416-427. doi: 10.1016/j.ymgme.2018.02.014. Epub 2018 Feb 28. Review. https://www.sciencedirect.com/science/article/pii/S1096719217307680?via%3Dihub Retrieved 12DEC19.

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