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Lessons Learned from HERA: the First Alport Syndrome Therapeutic Clinical Trial.

作者信息

Weinstock B André

机构信息

Alport Syndrome Foundation, Scottsdale, Arizona.

出版信息

Clin J Am Soc Nephrol. 2024 Aug 1;19(8):946-948. doi: 10.2215/CJN.0000000000000506. Epub 2024 Jun 21.

Abstract
摘要

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本文引用的文献

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A Randomized Controlled Clinical Trial Testing Effects of Lademirsen on Kidney Function Decline in Adults with Alport Syndrome.
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2
Polygenic risk alters the penetrance of monogenic kidney disease.
Nat Commun. 2023 Dec 14;14(1):8318. doi: 10.1038/s41467-023-43878-9.
3
Prevalence Estimates of Predicted Pathogenic Variants in a Population Sequencing Database and Their Implications for Alport Syndrome.
J Am Soc Nephrol. 2021 Sep;32(9):2273-2290. doi: 10.1681/ASN.2020071065. Epub 2021 Jun 18.
5
Diagnostic Utility of Exome Sequencing for Kidney Disease.
N Engl J Med. 2019 Jan 10;380(2):142-151. doi: 10.1056/NEJMoa1806891. Epub 2018 Dec 26.
7
Collagen COL4A3 knockout: a mouse model for autosomal Alport syndrome.
Genes Dev. 1996 Dec 1;10(23):2981-92. doi: 10.1101/gad.10.23.2981.

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