Novel therapeutic approaches to autosomal dominant polycystic kidney disease.

Autosomal dominant polycystic kidney disease (ADPKD) is an inherited disorder characterized by the progressive growth of renal cysts that, over time, destroy the architecture of the renal parenchyma and typically lead to kidney failure by the sixth decade of life. ADPKD is common and represents a leading cause of renal failure worldwide. Currently, there are no Food and Drug Administration-approved treatments for the disease, and the existing standard of care is primarily supportive in nature. However, significant advances in the understanding of the molecular biology of the disease have inspired investigation into potential new therapies. Several drugs designed to slow or arrest the progression of ADPKD have shown promise in preclinical models and clinical trials, including vasopressin receptor antagonists and somatostatin analogs. This article examines the literature underlying the rationale for molecular therapies for ADPKD and reviews the existing clinical evidence for their indication for human patients with the disease.
AuthorsWells B LaRiviere, Maria V Irazabal, Vicente E Torres
JournalTranslational research : the journal of laboratory and clinical medicine (Transl Res) Vol. 165 Issue 4 Pg. 488-98 (Apr 2015) ISSN: 1878-1810 [Electronic] United States
PMID25438190 (Publication Type: Journal Article, Review)
CopyrightCopyright © 2015 Elsevier Inc. All rights reserved.
  • Drug Design
  • Drug Discovery
  • Humans
  • Molecular Targeted Therapy
  • Polycystic Kidney, Autosomal Dominant (drug therapy)

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