Annemieke Aartsma‐Rus

Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

Judith C. van Deutekom, Anneke A. M. Janson, Ieke B. Ginjaar et al. · New England Journal of Medicine · 2007 · 760 citations

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

Catherine L. Bladen, David Salgado, Soledad Monges et al. · Human Mutation · 2015 · 717 citations · Full text

Dmd Mutations, Trial Planning, Engineering +12

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

Nathalie Goemans, M. Tulinius, Johanna T. van den Akker et al. · New England Journal of Medicine · 2011 · 659 citations · Full text

Digenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2

Richard J.L.F. Lemmers, Rabi Tawil, Lisa M. Petek et al. · Nature Genetics · 2012 · 641 citations · Full text

Rare Diseases, Developmental Biology, Mendelian Disorder +10

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

Annemieke Aartsma‐Rus, Arthur Μ. Krieg · Nucleic Acid Therapeutics · 2016 · 346 citations · Full text

Duchenne Muscular Dystrophy, Clinical Trials, Eteplirsen Saga +5