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Open access

Research article

First published 2023-12

Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy

Sarah Engelbeen, Daniel O'reilly, Davy van de Vijver [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2026-4

Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study

Emma T. Groenwold, Alicia Montulet, Tiberiu Stan [...]

Nucleic Acid Therapeutics

Free access

Research article

First published

Non-Ambulant Duchenne Patients Theoretically Treatable by Exon 53 Skipping have Severe Phenotype

Laurent Servais, Marie Montus, Caroline Le Guiner [...]

Journal of Neuromuscular Diseases

Open access

Article commentary

First published 2023-05-02

The Dilemma of Choice for Duchenne Patients Eligible for Exon 51 Skipping The European Experience

Annemieke Aartsma-Rus, Liesbeth De Waele, Saskia Houwen-Opstal [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023-06

Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy

Judith van Deutekom, Chantal Beekman, Suzanne Bijl [...]

Nucleic Acid Therapeutics

Open access

Research article

First published 2022

Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Paula R. Clemens, Vamshi K. Rao, Anne M. Connolly [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023-05-02

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2,Open-Label,4-Year Extension Study

Paula R. Clemens, Vamshi K. Rao, Anne M. Connolly [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2021

Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases

Yoshitsugu Aoki, Matthew J.A. Wood

Journal of Neuromuscular Diseases

Open access

Review article

First published 2024-04-30

Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis

E. Landfeldt, A. Alemán, S. Abner [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2022-02

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human,Multicenter,Two-Part,Open-Label,Phase 1/2 Trial

Laurent Servais, Eugenio Mercuri, Volker Straub [...]

Nucleic Acid Therapeutics

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