DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials

• Published in: Neurology Vol. 100; no. 15; p. e1540
• Main Authors: Muntoni, Francesco, Signorovitch, James, Sajeev, Gautam, Lane, Henry, Jenkins, Madeline, Dieye, Ibrahima, Ward, Susan J, McDonald, Craig, Goemans, Nathalie, Niks, Erik H, Wong, Brenda, Servais, Laurent, Straub, Volker, Guglieri, Michela, de Groot, Imelda J M, Chesshyre, Mary, Tian, Cuixia, Manzur, Adnan Y, Mercuri, Eugenio, Aartsma-Rus, Annemieke
• Format: Journal Article
• Language: English
• Published: United States 11.04.2023
• Subjects: Child; Genotype; Humans; Italy; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - genetics; Walking; Italy

Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients with untreated patients with the same genotype class. This avoids confounding of drug efficacy by genotype effects but also shrinks the pool of eligible controls, increasing ch...