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Clinical Trials and a View Toward the Future of ADPKD

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Cystogenesis

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 933))

Abstract

In light of the advances in the understanding of cystogenesis in clinical syndromes, potential therapeutic targets have been proposed. Among ciliopathies, autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary disease, and is characterized by the progressive enlargement of bilateral renal cysts, resulting in end-stage kidney failure. Progress in genetics and molecular pathobiology has enabled the development of therapeutic agents that can modulate aberrant molecular pathways. Recently, clinical trials using somatostatin analogs and vasopressin receptor antagonists were conducted, and resulted in the approval of tolvaptan in managing kidney disease in some countries. We will summarize the developments of therapeutic agents based on pathogenesis, and discuss recent findings in clinical trials. Moreover, issues such as the timing of the intervention and outcome assessment will be discussed.

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Acknowledgement

The authors would like to express appreciation for the great help of Professors Curie Ahn (Seoul National University Hospital) and Kyubeck Lee (Kangbuk Samsung Hospital), who have devoted themselves to managing patients with ADPKD over the past two decades. We would also like to extend our gratitude to Danang Sarang (www.pkdkorea.co.kr/), a group for patients with ADPKD in Korea, and the PKD Foundation International (http://www.pkdinternational.org/).

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Authors and Affiliations

  1. Department of Internal Medicine, Seoul National University Hospital, 101, Daehak-ro, Jongno-gu, Seoul, 03080, South Korea

    Hyunsuk Kim

  2. Department of Internal Medicine, Eulji General Hospital, 14, Hangeulbiseok-gil, Nowon-gu, Seoul, 01830, South Korea

    Young-Hwan Hwang

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  1. Hyunsuk Kim
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  2. Young-Hwan Hwang
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Correspondence to Hyunsuk Kim .

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Editors and Affiliations

  1. Department of Life systems, Sookmyung Women’s University, Seoul, Korea (Republic of)

    Jong Hoon Park

  2. Department of Internal Medicine, Seoul National University College of Medicine, Seoul, Korea (Republic of)

    Curie Ahn

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© 2016 Springer Science+Business Media Singapore

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Kim, H., Hwang, YH. (2016). Clinical Trials and a View Toward the Future of ADPKD. In: Park, J., Ahn, C. (eds) Cystogenesis. Advances in Experimental Medicine and Biology, vol 933. Springer, Singapore. https://doi.org/10.1007/978-981-10-2041-4_9

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