Elesclomol-copper therapy improves neurodevelopment in two children with Menkes disease

Por: Godoy-Molina E, Serrano NL, Jiménez-González A, Villaronga M, Marqués Pérez-Bryan RM, Varela-Fernández R, Lotz-Esquivel S, Hevia Tuñón A, Trivedi PP, Horn N, Standing JF, Mangas-Sanjuan V, Capdevila M, Mateos A, Broun D, Lutsenko S, Medina-Rivera IF, Artuch-Iriberri R, Jou-Munoz C, Roldan-Molina M, Pedro Arango Sancho, Saez-Villafañe M, Ortiz-de-Urbina JJ, Pieras-López A, Duero M, Rosa Farré Riba, Pijuan-Marquilles J, Hoenicka J, Sacchettini JC, Petris MJ, Gohil VM and Palau F

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Publicada: 1 oct 2025 Ahead of Print: 29 jul 2025

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Filiaciones:
Godoy-Molina E:
 Complex Chronic Children and Palliative Care Unit, Department of Pediatrics, Hospital Regional Universitario de Málaga, Málaga, Spain

Serrano NL:
 Laboratory of Neurogenetics and Molecular Medicine, Center for Genomic Scie, Institut de Recerca Sant Joan de Déu, Barcelona, Barcelona, Spain

Jiménez-González A:
 Department of Pediatrics, Complejo Asistencial Universitario de León, León, Spain

Villaronga M:
 Department of Pharmacy, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Marqués Pérez-Bryan RM:
 Centro de Atención Temprana Dr. Miguel de Linares Pezzi, Dulce Nombre de Maria Psychopedagogic Institute, Málaga, Spain

Varela-Fernández R:
 Department of Pharmacy, Complejo Asistencial Universitario de León, León, Spain

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 Laboratory of Neurogenetics and Molecular Medicine, Center for Genomic Scie, Institut de Recerca Sant Joan de Déu, Barcelona, Barcelona, Spain

Hevia Tuñón A:
 Department of Pediatrics, Complejo Asistencial Universitario de León, León, Spain

Trivedi PP:
 Department of Biochemistry & Biophysics, Texas A&M University, College Station, United States of America

Horn N:
 Department of Genetics, The Kennedy Centre and Copenhagen University Hospital, Rigshospital, Copenhagen, Denmark

Standing JF:
 Institute for Child Health, University College London, London, United Kingdom

Mangas-Sanjuan V:
 Department of Pharmacy and Pharmaceutical Technology and Parasitology, University of Valencia, Valencia, Spain

Capdevila M:
 Department of Chemistry, Faculty of Sciences, Universitat Autònoma de Barcelona, Cerdanyola de Vallès, Barcelona, Spain

Mateos A:
 Menkes International Association, Málaga, Spain

Broun D:
 Menkes International Association, Málaga, Spain

Lutsenko S:
 Department of Physiology, Johns Hopkins University School of Medicine, Baltimore, United States of America

Medina-Rivera IF:
 Neuropsychology Unit, Department of Pediatric Neurology, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Artuch-Iriberri R:
 Department of Clinical Biochemistry, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Jou-Munoz C:
 Department of Pathology, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Roldan-Molina M:
 Confocal Microscopy and Cellular Imaging Unit, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Pedro Arango Sancho:
 Department of Pediatric Nephrology, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Saez-Villafañe M:
 Department of Pharmacy, Complejo Asistencial Universitario de León, León, Spain

Ortiz-de-Urbina JJ:
 Department of Pharmacy, Complejo Asistencial Universitario de León, León, Spain

Pieras-López A:
 Department of Pharmacy, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Duero M:
 Department of Pharmacy, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Rosa Farré Riba:
 Department of Pharmacy, Hospital Sant Joan de Déu, Barcelona, Barcelona, Spain

Pijuan-Marquilles J:
 Laboratory of Neurogenetics and Molecular Medicine, Center for Genomic Scie, Institut de Recerca Sant Joan de Déu, Barcelona, Barcelona, Spain

Hoenicka J:
 Laboratory of Neurogenetics and Molecular Medicine, Center for Genomic Scie, Institut de Recerca Sant Joan de Déu, Barcelona, Barcelona, Spain

Sacchettini JC:
 Department of Biochemistry & Biophysics, Texas A&M University, College Station, United States of America

Petris MJ:
 Departments of Ophthalmology and Biochemistry, University of Missouri, Columbia, United States of America

Gohil VM:
 Department of Biochemistry & Biophysics, Texas A&M University, College Station, United States of America

Palau F:
 Laboratory of Neurogenetics and Molecular Medicine, Center for Genomic Scie, Institut de Recerca Sant Joan de Déu, Barcelona, Barcelona, Spain