Duchenne muscular dystrophy (DMD) is an X-linked progressive disorder associated with
muscle wasting and degeneration. The disease is caused by mutations in the gene that …

Muscular dystrophies (MDs) are a heterogeneous group of diseases of genetic origin
characterized by progressive skeletal muscle degeneration and weakness. There are …

This perspective article is concerned with the question of how proteomics, which is a core
technique of systems biology that is deeply embedded in the multi-omics field of modern …

Duchenne muscular dystrophy is a highly progressive muscle wasting disease of early
childhood and characterized by complex pathophysiological and histopathological changes …

Antisense oligonucleotide (ASO) has emerged as a promising therapeutic approach for
treating central nervous system (CNS) disorders by modulating gene expression with high …

Imetelstat is a novel, first‐in‐class, oligonucleotide telomerase inhibitor in development for
the treatment of hematologic malignancies including lower‐risk myelodysplastic syndromes …

Antisense-mediated exon skipping is one of the most promising therapeutic strategies for
Duchenne muscular dystrophy (DMD) and some antisense oligonucleotide (ASO) drugs …

Objective: This report summarizes the key discussions from the “Early Care (0–3 years) in
Duchenne Muscular Dystrophy” meeting, which aimed to address the challenges and …

Introduction Elevated transaminases and/or creatine phosphokinase can indicate underlying
muscle disease. Therefore, this study aims to determine the frequency of Duchenne …

Eteplirsen is indicated for treatment of exon 51 skip-amenable patients with DMD. Study
4658-102 (NCT03218995), a phase 2, open-label, dose-escalation trial, showed that …