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Introduction <p>Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder. Building on advancements in oligonucleotide chemistry and delivery, antisense oligonucleotides have emerged as a clinically approved therapeutic modality for treating Duchenne muscular dystrophy. However, the safety assessment of these drugs in clinical application remains limited.</p> Methods <p>This study utilized the FDA Adverse Event Reporting System to collect reports of adverse events related to four ASOs: Casimersen, Eteplirsen, Golodirsen, and Viltolarsen. The following four algorithms were used to quantify the adverse event (AE) signals: the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma-Poisson Shrinker (MGPS) models with Empirical Bayesian Geometric Mean (EBGM) estimation.</p> Results <p>Among 2,760 reports, the predominant AEs included respiratory infections, injection-site reactions, and systemic symptoms. A high reporting odds ratio was observed for poor venous access and product dose omission (Golodirsen, Eteplirsen, Casimersen). Proteinuria was reported for three ASOs (Viltolarsen, Eteplirsen, Casimersen). Among them,&#xa0;Viltolarsen exhibited the most potent signals (ROR = 55.41). Most AEs occurred more than 60 days post-dose (47–53%).</p> Conclusions <p>This study confirms the overall safety of four ASO drugs in line with previous publications. The adverse events, such as proteinuria, heart failure, and respiratory failure, highlight the importance of multidisciplinary management. However, limitations inherent to the FAERS database preclude the definitive establishment of causality and the accurate determination of incidence rates.</p>

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Systematic analysis of the adverse effects of used clinical antisense oligonucleotide drugs in DMD patients based on the FAERS database

  • 期刊论文

  • 发表日期：

    2026年1月19日

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  • Xiao-fang Zhang,
  • Wen-Guang Hu,
  • Jie Hu

摘要

Introduction

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder. Building on advancements in oligonucleotide chemistry and delivery, antisense oligonucleotides have emerged as a clinically approved therapeutic modality for treating Duchenne muscular dystrophy. However, the safety assessment of these drugs in clinical application remains limited.

Methods

This study utilized the FDA Adverse Event Reporting System to collect reports of adverse events related to four ASOs: Casimersen, Eteplirsen, Golodirsen, and Viltolarsen. The following four algorithms were used to quantify the adverse event (AE) signals: the Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma-Poisson Shrinker (MGPS) models with Empirical Bayesian Geometric Mean (EBGM) estimation.

Results

Among 2,760 reports, the predominant AEs included respiratory infections, injection-site reactions, and systemic symptoms. A high reporting odds ratio was observed for poor venous access and product dose omission (Golodirsen, Eteplirsen, Casimersen). Proteinuria was reported for three ASOs (Viltolarsen, Eteplirsen, Casimersen). Among them, Viltolarsen exhibited the most potent signals (ROR = 55.41). Most AEs occurred more than 60 days post-dose (47–53%).

Conclusions

This study confirms the overall safety of four ASO drugs in line with previous publications. The adverse events, such as proteinuria, heart failure, and respiratory failure, highlight the importance of multidisciplinary management. However, limitations inherent to the FAERS database preclude the definitive establishment of causality and the accurate determination of incidence rates.