[HTML][HTML] A phase 2 study of AMO-02 (Tideglusib) in congenital and childhood-onset myotonic dystrophy type 1 (DM1)
J Horrigan, TB Gomes, M Snape, N Nikolenko… - Pediatric Neurology, 2020 - Elsevier
Background GSK3β is an intracellular regulatory kinase that is dysregulated in multiple
tissues in type 1 myotonic dystrophy, a rare neuromuscular disorder that manifests at any
age. AMO-02 (tideglusib) inhibits GSK3β activity in preclinical models of type 1 myotonic
dystrophy and promotes cellular maturation as well as normalizes 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 …
tissues in type 1 myotonic dystrophy, a rare neuromuscular disorder that manifests at any
age. AMO-02 (tideglusib) inhibits GSK3β activity in preclinical models of type 1 myotonic
dystrophy and promotes cellular maturation as well as normalizes 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 …