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

• Главные авторы: Giulio Frontino (Автор), Tara Raouf (Автор), Daniele Canarutto (Автор), Eva Tirelli (Автор), Raffaella Di Tonno (Автор), Andrea Rigamonti (Автор), Maria Lucia Cascavilla (Автор), Cristina Baldoli (Автор), Roberta Scotti (Автор), Letizia Leocani (Автор), Su-Chun Huang (Автор), Franco Meschi (Автор), Graziano Barera (Автор), Vania Broccoli (Автор), Greta Rossi (Автор), Silvia Torchio (Автор), Raniero Chimienti (Автор), Riccardo Bonfanti (Автор), Lorenzo Piemonti (Автор)
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• Опубликовано: Frontiers Media S.A., 2021-12-01T00:00:00Z.
• Предметы: article
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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...