Gene-specific response to MuSK agonist antibody in the treatment of Congenital Myasthenic Syndromes
Kelly Ho, Ofosu Adjei-Afriyie, Ricardo Carmona-Martinez…·Hanns Lochmüller
This study tests a MuSK agonist antibody in Agrn-CMS and ColQ-CMS mouse models, showing benefit in Agrn-CMS but not ColQ-CMS, highlighting gene-specific treatment responses.