Therapeutic Approaches in Facioscapulohumeral Muscular Dystrophy
Justin Cohen(Yale University), Angela Lek(Muscular Dystrophy Association), Monkol Lek(Massachusetts General Hospital), Alec M. DeSimone(University of Massachusetts Chan Medical School)
Cited by 42
Related Papers
Exploring the phenotypic consequences of tissue specific gene expression variation inferred from GWAS summary statistics
|Nature Communications|2018|1.2k
Improving genetic diagnosis in Mendelian disease with transcriptome sequencing
|Science Translational Medicine|2017|805
Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne’s Muscular Dystrophy
|New England Journal of Medicine|2023|302