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Migalastat: A Review in Fabry Disease
Fabry disease is a rare lysosomal disorder characterized by deficient or absent α-galactosidase A activity resulting from mutations in the GLA gene. Migalastat (Galafold™), a pharmacological chaperone, stabilizes and facilitates trafficking of amenable mutant forms of α-galactosidase A enzyme from t...
Wedi'i Gadw mewn:
| Cyhoeddwyd yn: | Drugs |
|---|---|
| Prif Awduron: | , |
| Fformat: | Artigo |
| Iaith: | Inglês |
| Cyhoeddwyd: |
Springer International Publishing
2019
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| Pynciau: | |
| Mynediad Ar-lein: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6647464/ https://ncbi.nlm.nih.gov/pubmed/30875019 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40265-019-01090-4 |
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