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Repurposing ciclopirox as a pharmacological chaperone in a model of congenital erythropoietic porphyria
Author(s) -
Pedro Urquiza,
Ana Laín,
Arantza Sanz-Parra,
Jorge M. Charco,
Ganeko BernardoSeisdedos,
Pierre Dubus,
Esperanza González,
Virginia Gutiérrez-de-Juan,
Sandra García-Martínez,
Hasier Eraña,
Itxaso San Juan,
Iratxe Macías,
Fredj Ben Bdira,
Paula Pluta,
Gabriel Ortega,
Julen Oyarzábal,
Rosario GonzálezMuñiz,
Juan RodríguezCuesta,
Juan Anguíta,
Emilio Dı́ez,
JeanMarc Blouin,
Hubert de Verneuil,
José M. Mato,
Emmanuel Richard,
Juan Manuel FalcónPérez,
Joaquı́n Castilla,
Óscar Millet
Publication year - 2018
Publication title -
science translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 6.819
H-Index - 216
eISSN - 1946-6242
pISSN - 1946-6234
DOI - 10.1126/scitranslmed.aat7467
Subject(s) - antifungal , medicine , porphyria , pharmacology , dermatology
The off-patent marketed antifungal ciclopirox improves symptoms in a mouse model of congenital erythropoietic porphyria.

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