Dystrophin is a long rod-shaped protein that connects the subsarcolemmal cytoskeleton to a
complex of proteins in the surface membrane (dystrophin protein complex, DPC), with further …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Objective To continue evaluation of the long‐term efficacy and safety of eteplirsen, a
phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with …

Objective In prior open‐label studies, eteplirsen, a phosphorodiamidate morpholino
oligomer, enabled dystrophin production in Duchenne muscular dystrophy (DMD) with …

Introduction: Dystrophinopathy is a rare, severe muscle disorder, and nonsense mutations
are found in 13% of cases. Ataluren was developed to enable ribosomal readthrough of …

Background We report clinical safety and biochemical efficacy from a dose-ranging study of
intravenously administered AVI-4658 phosphorodiamidate morpholino oligomer (PMO) in …

Biomarkers for monitoring of disease progression and response to therapy are lacking for
muscle diseases such as Duchenne muscular dystrophy. Noninvasive in vivo molecular …

Background Local intramuscular administration of the antisense oligonucleotide PRO051 in
patients with Duchenne's muscular dystrophy with relevant mutations was previously …

Introduction: Duchenne muscular dystrophy (DMD) subjects≥ 5 years with nonsense
mutations were followed for 48 weeks in a multicenter, randomized, double‐blind, placebo …

Introduction: An international clinical trial enrolled 174 ambulatory males≥ 5 years old with
nonsense mutation Duchenne muscular dystrophy (nmDMD). Pretreatment data provide …