Wolfram syndrome type 1 research hub

Towards a personalized precision medicine in rare disease: tirzepatide (a dual glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist) monotherapy in patients with Wolfram syndrome type 1.

This study is testing a new medicine called Mounjaro for people with Wolfram syndrome type 1. It's looking to see if the medicine helps the body make more of a natural substance called C-peptide, which is important for managing blood sugar. This is an early-stage study to understand how well it works.