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Fabry Disease Therapy: State-of-the-Art and Current Challenges
Fabry disease (FD) is a lysosomal storage disorder caused by mutations of the GLA gene that lead to a deficiency of the enzymatic activity of α-galactosidase A. Available therapies for FD include enzyme replacement therapy (ERT) (agalsidase alfa and agalsidase beta) and the chaperone migalastat. Des...
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| 出版年: | Int J Mol Sci |
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| 主要な著者: | , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
MDPI
2020
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7794923/ https://ncbi.nlm.nih.gov/pubmed/33379210 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22010206 |
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