A Phase 2 Study of AMO-02 (tideglusib) in Congenital and Childhood Onset Myotonic Dystrophy Type 1 (DM1)

GSK3 β is an intracellular regulatory kinase that is dysregulated in multiple tissues in Type 1 myotonic dystrophy (DM1), a rare neuromuscular disorder that manifests at any age. AMO-02 (tideglusib) inhibits GSK3β activity in preclinical models of DM1 and promotes cellular maturation as well as normali zing aberrant molecular and behavioral phenotypes. This Phase 2 study assessed the pharmacokinetics, safety and tolerability, and preliminary efficacy, of AMO-02 in adolescents and adults with Congenital and Childhood-onset DM1.

Source: Pediatric Neurology - August 3, 2020 Category: Neurology Authors: Joseph Horrigan, Tiago Bernardino Gomes, Mike Snape, Nikoletta Nikolenko, Alison McMorn, Stuart Evans, Alex Yaroshinsky, Oscar Della Pasqua, Sean Oosterholt, Hanns Lochm üller Tags: Research Paper Source Type: research