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

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

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...

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Main Authors: Patrick Deegan, Özlem Göker-Alpan, Tarekegn Geberhiwot, Robert J. Hopkin, Elena Lukina, Anna Tylki‐Szymańska, Atef Zaher, Charlotte Sensinger, Sebastiaan J.M. Gaemers, Vijay Modur, Beth L. Thurberg, Jyoti Sharma, Behzad Najafian, Michael Mauer, Pronabesh DasMahapatra, William R. Wilcox, Dominique P. Germain
Format: Artigo
Language:English
Published: 2022
Online Access:https://doi.org/10.1016/j.ymgme.2022.11.002
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https://doi.org/10.1016/j.ymgme.2022.11.002

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