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

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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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
Series:Scientific Reports
Subjects:
Cantú syndrome
Cardiac hypertrophy
Drug repurposing
Hypertrichosis
Sulfonylurea
Online Access:https://doi.org/10.1038/s41598-025-00547-9
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https://doi.org/10.1038/s41598-025-00547-9

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