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Updated: Mar 13, 2026

Use of Ultra-high Field MRI in Small Rodent Models of Polycystic Kidney Disease for In Vivo Phenotyping and Drug Monitoring
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Clinical Trials and a View Toward the Future of ADPKD

Hyunsuk Kim1, Young-Hwan Hwang2

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

Advances in Experimental Medicine and Biology
|October 13, 2016
PubMed

Insights

Autosomal dominant polycystic kidney disease (ADPKD) treatments are advancing. New therapies targeting cystogenesis pathways, like tolvaptan, show promise for managing this common inherited kidney disease.

Area of Science:

  • Nephrology
  • Genetics
  • Molecular Biology

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) is a prevalent genetic disorder.
  • It leads to progressive kidney cyst development and eventual kidney failure.
  • Understanding cystogenesis has identified new therapeutic targets.

Purpose of the Study:

  • To review therapeutic agents for ADPKD based on disease pathogenesis.
  • To discuss recent clinical trial findings for ADPKD treatments.
  • To explore intervention timing and outcome assessment in ADPKD management.

Main Methods:

  • Literature review of ADPKD pathogenesis.
  • Analysis of clinical trial data for somatostatin analogs and vasopressin receptor antagonists.
  • Discussion of therapeutic strategies and outcome measures.

Main Results:

  • Progress in understanding ADPKD has led to targeted therapies.
  • Tolvaptan, a vasopressin receptor antagonist, is approved in some regions.
  • Clinical trials are evaluating various agents modulating aberrant molecular pathways.

Conclusions:

  • Therapeutic options for ADPKD are expanding based on molecular insights.
  • Tolvaptan represents a significant advancement in managing ADPKD.
  • Further research is needed on optimal intervention timing and outcome assessment.
Keywords:
Autosomal dominantClinical trialsCystogenesisPolycystic kidneyTherapeutics

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