Associations between dystrophin genotype and ambulatory outcomes in DMD: implications for trials of genotype-targeted therapies
Associations between dystrophin genotype and ambulatory outcomes in DMD: implications for trials of genotype-targeted therapies
This study evaluated the suitability of genotypically unmatched controls in DMD by quantifying genotype effects on 1-year changes in North Star Ambulatory Assessment (NSAA) total score across data sources.
This late-breaking poster was presented at WMS 2021.
Authors: F. Muntoni, J. Signorovitch, G. Sajeev, H. Lane, M. Jenkins, I. Dieye, S. Ward, C. McDonald, N. Goemans, E. Niks, B. Wong, L. Servais, V. Straub, I. De Groot, C. Tian, A. Manzur, and E. Mercuri.