Eteplirsen: First Global Approval

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

The case for eteplirsen: Paving the way for precision medicine

(2016) M. Carrie Miceli et al.   MOLECULAR GENETICS AND METABOLISM

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Dystromirs as Serum Biomarkers for Monitoring the Disease Severity in Duchenne Muscular Dystrophy

(2013) Irina T. Zaharieva et al.   PLoS One

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Safety Pharmacology and Genotoxicity Evaluation of AVI-4658

(2010) Peter Sazani et al.   INTERNATIONAL JOURNAL OF TOXICOLOGY

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY