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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

Free access

Research article

First published 2020

Clinical Phenotypes of DMD Exon 51 Skip Equivalent Deletions: A Systematic Review

Megan A. Waldrop, Rabah Ben Yaou, Karin K. Lucas [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2025-04

Targeting a Novel Site in Exon 51 with Antisense Oligonucleotides Induces Enhanced Exon Skipping in a Mouse Model of Duchenne Muscular Dystrophy

Todd Oppeneer, Yulan Qi, Joshua Henshaw [...]

Nucleic Acid Therapeutics

Open access

Research article

First published 2025-04

BMN 351-Induced Exon Skipping and Dystrophin Expression in Skeletal and Cardiac Muscle Lead to Preservation of Motor Function in a Mouse Model of Exon 51 Skip-Amenable Duchenne Muscular Dystrophy

Todd Oppeneer, Yulan Qi, Joshua Henshaw [...]

Nucleic Acid Therapeutics

Open access

Research article

First published 2021

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial

Craig M. McDonald, Perry B. Shieh, Hoda Z. Abdel-Hamid [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2007-09

Comparative Analysis of Antisense Oligonucleotide Sequences for Targeted Skipping of Exon 51 During Dystrophin Pre-mRNA Splicing in Human Muscle

V. Arechavala-Gomeza, I.R. Graham, L.J. Popplewell [...]

Human Gene Therapy

Retricted access

Research article

First published 2012-10

Exon Skipping Quantification by Quantitative Reverse-Transcription Polymerase Chain Reaction in Duchenne Muscular Dystrophy Patients Treated with the Antisense Oligomer Eteplirsen

Karen Anthony, Lucy Feng, Virginia Arechavala-Gomeza [...]

Human Gene Therapy Methods (Part B)

Open access

Research article

First published 2014

Prolonged Ambulation in Duchenne Patients with a Mutation Amenable to Exon 44 Skipping

J.C. van den Bergen, H.B. Ginjaar, E.H. Niks [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2017-10

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

Annemieke Aartsma-Rus, Volker Straub, Robert Hemmings [...]

Nucleic Acid Therapeutics

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