Case Report: Off-Label Liraglutide Use in Children With Wolfram Syndrome Type 1: Extensive Characterization of Four Patients

Aims: Wolfram syndrome type 1 is a rare recessive monogenic form of insulin-dependent diabetes mellitus with progressive neurodegeneration, poor prognosis, and no cure. Based on preclinical evidence we hypothesized that liraglutide, a glucagon-like peptide-1 receptor agonist, may be repurposed for t...

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Detalles Bibliográficos
Autores principales: Frontino, Giulio, Raouf, Tara, Canarutto, Daniele, Tirelli, Eva, Di Tonno, Raffaella, Rigamonti, Andrea, Cascavilla, Maria Lucia, Baldoli, Cristina, Scotti, Roberta, Leocani, Letizia, Huang, Su-Chun, Meschi, Franco, Barera, Graziano, Broccoli, Vania, Rossi, Greta, Torchio, Silvia, Chimienti, Raniero, Bonfanti, Riccardo, Piemonti, Lorenzo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Pediatrics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8712700/
https://www.ncbi.nlm.nih.gov/pubmed/34970515
http://dx.doi.org/10.3389/fped.2021.755365

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8712700/
https://www.ncbi.nlm.nih.gov/pubmed/34970515
http://dx.doi.org/10.3389/fped.2021.755365

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