Article
DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.
Neurology - 11 Apr 2023
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
Abstract excerpt
BACKGROUND AND OBJECTIVES: Clinical trials of genotype-targeted treatments in Duchenne muscular dystrophy (DMD) traditionally compare treated patients with untreated patients with the same DMD genotype class. This avoids confounding of drug efficacy by genotype effects but also shrinks the pool of eligible controls, increasing challenges for trial enrollment in this already rare disease. To evaluate the...
Topics
- Child
- Humans
- Muscular Dystrophy, Duchenne
- Genotype
- Walking
- Italy