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Open access

Research article

First published 2020-04

Systemic Evaluation of Chimeric LNA/2′-O-Methyl Steric Blockers for Myotonic Dystrophy Type 1 Therapy

Melina Christou, Jesper Wengel, Kleitos Sokratous [...]

Nucleic Acid Therapeutics

Free access

Research article

First published 2018

Prenatal,Neonatal,and Early Childhood Features in Congenital Myotonic Dystrophy

Eugenio Zapata-Aldana, Delia Ceballos-Sáenz, Rhiannon Hicks [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2023

Resistance Exercise Training Rescues Mitochondrial Dysfunction in Skeletal Muscle of Patients with Myotonic Dystrophy Type 1

Valeria Di Leo, Conor Lawless, Marie-Pier Roussel [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2023-05

Advances in CRISPR/Cas9 Genome Editing for the Treatment of Muscular Dystrophies

Sina Fatehi, Ryan M. Marks, Matthew J. Rok [...]

Human Gene Therapy

Open access

Research article

First published 2021

Clinical Outcome Evaluations and CBT Response Prediction in Myotonic Dystrophy

Daniël van As, Kees Okkersen, Guillaume Bassez [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024-04-30

Treatment Approaches for Altered Facial Expression: A Systematic Review in Facioscapulohumeral Muscular Dystrophy and Other Neurological Diseases

Nathaniël B. Rasing, Willianne A. van de Geest-Buit, On Ying A. Chan [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2023-07-04

RNA Transcript Diversity in Neuromuscular Research

Wooi Fang Lim, Carlo Rinaldi

Journal of Neuromuscular Diseases

Open access

Review article

First published 2021

Modifiers of CAG/CTG Repeat Instability: Insights from Mammalian Models

Vanessa C. Wheeler, Vincent Dion

Journal of Huntington's Disease

Retricted access

Research article

First published 2015-08

Generation of Human Induced Pluripotent Stem Cells from Extraembryonic Tissues of Fetuses Affected by Monogenic Diseases

Paola Spitalieri, Rosa V. Talarico, Annalisa Botta [...]

Cellular Reprogramming

Retricted access

Research article

First published 2022-08

Enhanced Delivery of Ligand-Conjugated Antisense Oligonucleotides (C16-HA-ASO) Targeting Dystrophia Myotonica Protein Kinase Transcripts for the Treatment of Myotonic Dystrophy Type 1

Siham Ait Benichou, Dominic Jauvin, Thiéry De Serres-Bérard [...]

Human Gene Therapy

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