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Treatment of autosomal dominant polycystic kidney disease (ADPKD): the new horizon for children with ADPKD

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Abstract

Polycystic kidney disease (PKD) is the most common inherited renal disorder. Patients with PKD remain clinically asymptomatic for decades, while significant anatomic and physiologic systemic changes take place. Sequencing of the responsible genes and identification of their protein products have significantly expanded our understanding of the pathophysiology of PKD. The molecular basis for cystogenesis is being unraveled, leading to new targets for therapy and giving hope to millions of people suffering from PKD. This has direct implications for children with PKD with regard to screening for the disease and identification of high-risk individuals. In this article we provide a review of the clinical manifestations in children with autosomal dominant polycystic kidney disease (ADPKD), the genetic and molecular basis for the disease, and a concise review of potential therapies being evaluated.

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  1. Emory School of Medicine, VA Medical Center, 1670 Clairmont Road, mail code 111, Decatur, GA, 30033, USA

    Dana Rizk

  2. Emory University Hospital, General Clinical Research Center, Decatur, GA, USA

    Arlene Chapman

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  1. Dana Rizk
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  2. Arlene Chapman
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Correspondence to Dana Rizk.

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Rizk, D., Chapman, A. Treatment of autosomal dominant polycystic kidney disease (ADPKD): the new horizon for children with ADPKD. Pediatr Nephrol 23, 1029–1036 (2008). https://doi.org/10.1007/s00467-007-0706-9

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  • DOI: https://doi.org/10.1007/s00467-007-0706-9

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