טוען...
In Vitro and In Vivo Amenability to Migalastat in Fabry Disease
Migalastat (1-deoxygalactonojirimycin) is approved for the treatment of Fabry disease (FD) in patients with an amenable mutation. Currently, there are at least 367 amenable and 711 non-amenable mutations known, based on an in vitro good laboratory practice (GLP) assay. Recent studies demonstrated th...
שמור ב:
| הוצא לאור ב: | Mol Ther Methods Clin Dev |
|---|---|
| Main Authors: | , , |
| פורמט: | Artigo |
| שפה: | Inglês |
| יצא לאור: |
American Society of Gene & Cell Therapy
2020
|
| נושאים: | |
| גישה מקוונת: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7490640/ https://ncbi.nlm.nih.gov/pubmed/32995357 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.08.012 |
| תגים: |
הוספת תג
אין תגיות, היה/י הראשונ/ה לתייג את הרשומה!
|