Genotype-unmatched controls are feasible for drug development in Duchenne Muscular Dystrophy (DMD)
Genotype-unmatched controls are feasible for drug development in Duchenne Muscular Dystrophy (DMD)
This study evaluated the prospect of using genotype-unmatched controls when 1) DMD genotype class effects are small and precisely known and 2) stronger baseline prognostic factors are accounted for in a hypothetical clinical trial for effects on 1-year change in the North Star Ambulatory Assessment total score (ΔNSAA).
This poster was presented at WMS 2022 and MDA 2023.
Authors: F. Muntoni, J. Signorovitch, M. Frean, M. Fillbrunn, G. Sajeev, S. Ward, C. McDonald, N. Goemans, E. Niks, B. Wong, L. Servais, V. Straub, I. de Groot, M. Chesshyre, C. Tian, A. Manzur, E. Mercuri, A. Aartsma-Rus, and Study Groups.