Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study.

PB, Deegan; O, Goker-Alpan; T, Geberhiwot; RJ, Hopkin; E, Lukina; A, Tylki-Szymanska; A, Zaher; C, Sensinger; SJM, Gaemers; V, Modur; BL, Thurberg; J, Sharma; B, Najafian; M, Mauer; P, DasMahapatra; WR, Wilcox; DP, Germain

doi:10.1016/j.ymgme.2022.11.002

Public

Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study.

Shared by NobleBlocks on Nov 9, 2022 • 12:00 AM UTC

Authors:

Deegan PB

Goker-Alpan O

Geberhiwot T

Abstract

Venglustat inhibits the enzymatic conversion of ceramide to glucosylceramide, reducing available substrate for the synthesis of more complex glycosphingolipids. It offers a potential new approach to the treatment of patients with Fabry disease (α-Gal A deficiency), in whom progressive accumulation o...

Subject

Fabry Disease

Substrate Reduction Therapy

Lysosomal Storage Disorder

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