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

• Main Authors: Giulio Frontino (Author), Tara Raouf (Author), Daniele Canarutto (Author), Eva Tirelli (Author), Raffaella Di Tonno (Author), Andrea Rigamonti (Author), Maria Lucia Cascavilla (Author), Cristina Baldoli (Author), Roberta Scotti (Author), Letizia Leocani (Author), Su-Chun Huang (Author), Franco Meschi (Author), Graziano Barera (Author), Vania Broccoli (Author), Greta Rossi (Author), Silvia Torchio (Author), Raniero Chimienti (Author), Riccardo Bonfanti (Author), Lorenzo Piemonti (Author)
• Format: Book
• Published: Frontiers Media S.A., 2021-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...