Golodirsen

Last Updated: 02/24/2026

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Found 69 publications

Small RNA or oligonucleotide drugs and challenges in evaluating drug-drug interactions.

Small RNA or oligonucleotide drugs and challenges in evaluating drug-drug interactions.

Journal: Frontiers in pharmacology

Published: October 07, 2025

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.

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

In Vivo Evaluation of Multiple Exon Skipping with Peptide-PMOs in Cardiac and Skeletal Muscles in Dystrophic Dogs.

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.

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

Systemic Injection of Peptide-PMOs into Humanized DMD Mice and Detection by RT-PCR and ELISA.

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

Tips to Design Effective Splice-Switching Antisense Oligonucleotides for Exon Skipping and Exon Inclusion.

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

Quantitative Evaluation of Exon Skipping in Immortalized Muscle Cells in Vitro.

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.

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.

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

An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.

An Overview of Recent Advances and Clinical Applications of Exon Skipping and Splice Modulation for Muscular Dystrophy and Various Genetic Diseases.

Journal: Methods in molecular biology (Clifton, N.J.)

Published: July 28, 2025

Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis.

Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis.

Journal: Journal of comparative effectiveness research

Published: July 18, 2025

Antisense oligonucleotide as novel therapies for neurogenetic disorders

Antisense oligonucleotide as novel therapies for neurogenetic disorders

Journal: Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics

Published: January 08, 2025

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Last Updated: 02/24/2026