A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease

Ashe, Karen M.; Marshall, John; Budman, Eva; Bangari, Dinesh S.; Nietupski, Jennifer B.; Desnick, Robert J.; Scheule, Ronald K.; Leonard, John P.; Cheng, Seng H.

doi:10.1016/j.ymgme.2012.11.026

Molecular Genetics and Metabolism

2013

DOI: 10.1016/j.ymgme.2012.11.026

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A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease

Karen M. Ashe¹,

John Marshall²,

Eva Budman³

et al.

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A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease

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