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

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