Jimenez-Moreno, Aura Cecilia; Nikolenko, Nikoletta; Kierkegaard, Marie; Blain, Alasdair P.; Newman, Jane; Massey, Charlotte; Moat, Dionne; Sodhi, Jas; Atalaia, Antonio; Gorman, Grainne S.; Turner, Chris; Lochmüller, Hanns
(Wiley, 2019)
Objectives: Defining clinically relevant outcome measures for myotonic dystrophy type 1 (DM1) that can be valid and feasible for different phenotypes has proven problematic. The Outcome Measures for Myotonic Dystrophy ...