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Last Updated: 10/31/2025
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Found 174 publications
The Utilization, Reimbursement, and Cost of Targeted Therapies for Duchenne Muscular Dystrophy (DMD) in US Medicaid Programs: A Descriptive Trend Analysis from 2017 to 2022.
Journal: Pharmaceutical medicine
Published: September 09, 2025
Association between exon-skipping therapy with eteplirsen and cardiac outcomes in Duchenne muscular dystrophy.
Journal: Journal of neuromuscular diseases
Published: August 20, 2025
In Vivo Evaluation of Single- and Multi-Exon-Skipping in mdx52 Mice.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Evolution and Breakthroughs in Exon Skipping and Splice Modulation: From Inception to Clinical Success.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Detection by RT-PCR and ELISA.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells in Vitro.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Creation of DMD Muscle Cell Model Using CRISPR-Cas9 Genome Editing to Test the Efficacy of Antisense-Mediated Exon Skipping.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Direct Reprogramming of Human DMD Fibroblasts into Myotubes for In Vitro Evaluation of Antisense-Mediated Exon Skipping and Exons 45-55 Skipping Accompanied by Rescue of Dystrophin Expression.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Progress and Future Directions in Exon Skipping and Inclusion Therapies: The Landscape of Oligonucleotide-Based Genetic Medicine.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Exon-Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy.
Journal: Methods in molecular biology (Clifton, N.J.)
Published: July 28, 2025
Last Updated: 10/31/2025