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Cystinosis: Therapy adherence and metabolic monitoring in patients treated with immediate-release cysteamine
BACKGROUND: Cystinosis is a metabolic disease caused by intracellular accumulation of cystine within lysosomes. Development of symptoms can be delayed significantly by a life-long therapy with cysteamine, a drug that enters the lysosome and reacts with cystine thereby enabling its export from the or...
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| 發表在: | Mol Genet Metab Rep |
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| Main Authors: | , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
Elsevier
2020
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7358454/ https://ncbi.nlm.nih.gov/pubmed/32685378 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2020.100620 |
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