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...
Saved in:
| Main Authors: | , , , , , , , , , , , , , , , , |
|---|---|
| 格式: | Artigo |
| 语言: | 英语 |
| 出版: |
2022
|
| 在线阅读: | https://doi.org/10.1016/j.ymgme.2022.11.002 |
| 标签: |
添加标签
没有标签, 成为第一个标记此记录!
|