Therapeutic Potential for CFTR Correctors in Autosomal Recessive Polycystic Kidney DiseaseSummary
Background & Aims: Autosomal recessive polycystic kidney disease (ARPKD) is caused by mutations in PKHD1, encoding fibrocystin/polyductin (FPC). Severe disease occurs in perinates. Those who survive the neonatal period face a myriad of comorbidities, including systemic and portal hypertensio...
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Main Authors: | , , |
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Format: | article |
Language: | EN |
Published: |
Elsevier
2021
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Subjects: | |
Online Access: | https://doaj.org/article/e684dd6174c740629decf06a8b20f547 |
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