Treatment of overactive KATP channels with glibenclamide in a zebrafish model and a clinical trial in humans with Cantú syndrome

Abstract This study explores the efficacy of glibenclamide, a KATP channel inhibitor, for treating Cantú syndrome (CS), a genetic disorder characterized by hypertrichosis and cardiovascular abnormalities. Treatment with glibenclamide for Cantú syndrome has only been reported in a single case report....

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Bibliographic Details

Published in:	Scientific Reports
Main Authors:	Lotte Kleinendorst, Sarah E. Siegelaar, Helen I. Roessler, Lema Meiwand, Malou van den Boogaard, Rianne H.A.C.M. de Bruin-Bon, Kirsten F. van Duinen, R. Nils Planken, Elisabeth H. Jaspars, Patrick M.J.H. Kemperman, Berto J. Bouma, Colin G. Nichols, Marcel W. Bekkenk, Gijs W. van Haaften, Mieke M. van Haelst
Format:	Article
Language:	English
Published:	Nature Portfolio 2025-05-01
Subjects:	Cantú syndrome Cardiac hypertrophy Drug repurposing Hypertrichosis Sulfonylurea
Online Access:	https://doi.org/10.1038/s41598-025-00547-9