Fabry disease


Can I combine ERT with other Fabry disease treatments like Galafold?

Theoretically combining chaperone therapy with enzyme replacement therapy (ERT) should stabilize the ERT enzyme and help it enter the lysosome to breakdown stored material. However, the timing, safety, and effectiveness of treating Fabry disease with both ERT and chaperone therapy has not been established. In addition, access to both could be difficult as insurance will probably not pay for both medications to be used in one individual as it would be considered experimental. Always speak with your Fabry disease specialist before starting or switching to a new treatment.

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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