Expert Opinion on Drug Safety最新文献_第2页

Influence factors of metronidazole-related CNS disorders: an analysis of the Japan adverse drug event report and FDA adverse event reporting system.

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-03 DOI: 10.1080/14740338.2025.2486308

Keisuke Takada, Yuki Enoki, Masaru Samura, Yuki Igarashi, Kazuaki Taguchi, Koji Tanikawa, Kazuaki Matsumoto

{"title":"Influence factors of metronidazole-related CNS disorders: an analysis of the Japan adverse drug event report and FDA adverse event reporting system.","authors":"Keisuke Takada, Yuki Enoki, Masaru Samura, Yuki Igarashi, Kazuaki Taguchi, Koji Tanikawa, Kazuaki Matsumoto","doi":"10.1080/14740338.2025.2486308","DOIUrl":"10.1080/14740338.2025.2486308","url":null,"abstract":"Background: Metronidazole (MNZ) can be administered for various infections. The impact of comorbidities/concomitant drugs on MNZ-induced central nervous system (CNS) disorders remains unclear.Research design and methods: We assessed the risk of metronidazole-related CNS disorders using the Japan Adverse Drug Event Report (JADER, May 2023) and the US Food and Drug Administration Adverse Event Reporting System (FAERS, Q1 2023), excluding comorbidities/concomitant drugs. Clonazepam and diazepam were evaluated as potential prophylactics based on the efficacy of benzodiazepines for MNZ-related CNS disorders. Reporting odds ratios (ROR) and 95% confidence intervals (CI) were calculated. Additionally, sensitivity analysis by sex and age was conducted.Results: The ROR (95% CI) of CNS disorders associated with MNZ in JADER and FAERS were 3.16 (2.69-3.72) and 1.69 (1.64-1.73), respectively. MNZ was significantly related to CNS disorders after excluding comorbidities (brain/spinal cord or liver abscesses) and concomitant drugs (glucocorticoids, antiepileptic, antiparkinson, and schizophrenia drugs). In sensitivity analysis, MNZ was significantly related to CNS disorders, despite sex and age. The ROR in the concomitant with clonazepam (CZP) was 0.70 (0.53-0.92) in FAERS.Conclusion: MNZ may be associated with CNS disorders, even if comorbidities/concomitant drugs that are potential risk factors for CNS disorders are excluded. Additionally, CZP may suppress CNS disorders.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"1-7"},"PeriodicalIF":3.0,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Real-world data analysis of topotecan in combination with Bevacizumab or CycloPhosphamide in the FDA adverse event reporting system (FAERS) database.

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-02 DOI: 10.1080/14740338.2025.2488240

Huihui Chen, Guobiao Zhuang, Shihao Hong, Jing Wu

{"title":"Real-world data analysis of topotecan in combination with Bevacizumab or CycloPhosphamide in the FDA adverse event reporting system (FAERS) database.","authors":"Huihui Chen, Guobiao Zhuang, Shihao Hong, Jing Wu","doi":"10.1080/14740338.2025.2488240","DOIUrl":"https://doi.org/10.1080/14740338.2025.2488240","url":null,"abstract":"Background: The main purpose of this study is to observe and detect adverse reactions to the combination of topotecan, bevacizumab and cyclophosphamide, to learn more about possible adverse drug reactions (ADRs) and to help doctors make the right medication decisions and treatment plans.Research design and methods: Adverse event signals were detected and quantified using data from the U.S. Food and Drug Administration's Adverse Event Reporting System using reporting ratios, proportions of reports (PRR), Bayesian Confidence Propagation Neural Networks (BCPN), and empirical Bayesian Geometric Mean (EBGM). Subgroup analyses were performed to compare adverse events associated with topotecan alone.Results: The analysis of FAERS data revealed a total of 1,789 primary suspected adverse events (PS AEs) linked to topotecan. The Weibull shape parameter (β) for females was lower than for males across all age groups, indicating a potentially higher susceptibility to the adverse effects of topotecan in female patients.Conclusions: This study proved several expected and new adverse drug reactions associated with the combination of topotecan, bevacizumab, and cyclophosphamide. While some ADRs, such as neutropenia and anemia, align with the known adverse profile of topotecan, the detection of novel signals, including potential gender-based differences in drug response, warrants further investigation.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"1-12"},"PeriodicalIF":3.0,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143763307","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Adverse events of celecoxib associated with the central nervous system and cancer: a disproportionality analysis of the FDA adverse event reporting system.

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-02 DOI: 10.1080/14740338.2025.2487140

Yikuan Du, Mengting Zhang, Mianhai Chen, Mianda Hu, Wenqi Zeng, Xiaolin Cai, Weichui Zhang, Jinfeng Zhu, Mingjun Zhong, Yi Liu, Chun Yang

{"title":"Adverse events of celecoxib associated with the central nervous system and cancer: a disproportionality analysis of the FDA adverse event reporting system.","authors":"Yikuan Du, Mengting Zhang, Mianhai Chen, Mianda Hu, Wenqi Zeng, Xiaolin Cai, Weichui Zhang, Jinfeng Zhu, Mingjun Zhong, Yi Liu, Chun Yang","doi":"10.1080/14740338.2025.2487140","DOIUrl":"10.1080/14740338.2025.2487140","url":null,"abstract":"Background: Celecoxib is now clinically recognized as a candidate for treating various neurological disorders and cancers. The recent emergence of some serious adverse reactions is concerning.Research design and methods: We carried out data mining on the FDA Adverse Event Reporting System (FAERS) for adverse events (AEs) with celecoxib as the main suspect drug and conducted a disproportionality analysis.Results: 111,155,092 AE reports were extracted from FAERS, and 32,841 AEs with celecoxib as the primary suspected drug were identified. Celecoxib AEs were predominantly reported in cardiac disorders (n = 9602) and nervous system disorders (n = 4045). Cerebrovascular accident (n = 3109, PRR = 3.24) ranked second in the number of reports and cerebrovascular disorder (n = 265, PRR = 5.06) ranked second in signal intensity, was described as rare in the instructions. Nine unexpected and serious AEs were discovered, such as Stevens-Johnson syndrome (n = 175, IC025 = 1.7), breast disease male (n = 4, IC025 = 1.54), and squamous cell carcinoma of the head and neck (n = 4, IC025 = 0.96). At 200 mg, celecoxib was more linked to musculoskeletal and connective AEs; At 400 mg, it was more linked to neurological and cardiovascular AEs.Conclusions: Unexpected AEs of celecoxib in neurological diseases and cancer have been identified, offering valuable insights for monitoring and risk assessment in future clinical applications.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"1-12"},"PeriodicalIF":3.0,"publicationDate":"2025-04-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751691","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Comparison of the pharmacovigilance signals of cardiac and renal adverse events associated with sacubitril/valsartan and valsartan alone based on the FAERS database. 基于FAERS数据库的沙比利/缬沙坦与缬沙坦单用与心脏和肾脏不良事件相关的药物警戒信号比较

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-01 Epub Date: 2024-12-03 DOI: 10.1080/14740338.2024.2436100

Zhenhui Chen, Jingwen Li, Yan Zhou, Qining Qiu, Dapeng Yan, Gang Peng, Yongpei Xu, Yanrong Ye, Yun Shen

{"title":"Comparison of the pharmacovigilance signals of cardiac and renal adverse events associated with sacubitril/valsartan and valsartan alone based on the FAERS database.","authors":"Zhenhui Chen, Jingwen Li, Yan Zhou, Qining Qiu, Dapeng Yan, Gang Peng, Yongpei Xu, Yanrong Ye, Yun Shen","doi":"10.1080/14740338.2024.2436100","DOIUrl":"10.1080/14740338.2024.2436100","url":null,"abstract":"Background: In clinical practice, cardiovascular and renal safety profiles of sacubitril/valsartan, compared with those of valsartan alone, remain controversial. Therefore, we aimed to compare the pharmacovigilance signals related to cardiovascular and renal adverse events between sacubitril/valsartan and valsartan alone using the Food and Drug Administration Adverse Event Reporting System (FAERS) database.Research design and methods: Raw report data on cardiac and renal adverse events associated with sacubitril/valsartan and valsartan alone were retrieved from the FAERS database using preferred terms from the Medical Dictionary for Regulatory Activities. Preferred terms were mapped to System Organ Classes, and the time to onset of adverse events associated with sacubitril/valsartan and valsartan alone was calculated.Results: Most adverse events associated with sacubitril/valsartan occurred within the first month, whereas adverse events were more prevalent 6 months to 1 year after administration of valsartan alone. Adverse events reported for sacubitril/valsartan and valsartan alone included cardiac failure, cardiogenic shock, and ventricular fibrillation. Considering sacubitril/valsartan only, adverse events reported were renal impairment, renal failure, and acute kidney injury. For valsartan alone, adverse events reported included arrhythmia and angina pectoris.Conclusions: Sacubitril/valsartan carries a higher renal safety risk and lower cardiac safety risk than valsartan alone.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"445-452"},"PeriodicalIF":3.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142767592","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

New strategies to manage the safety of cladribine in patients with multiple sclerosis. 多发性硬化症患者克拉德滨安全性管理的新策略。

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-01 Epub Date: 2025-01-03 DOI: 10.1080/14740338.2024.2448826

Paola Reitano, Clara G Chisari, Francesco Patti

引用次数: 0

Drug classes associated with the development of fulminant type 1 diabetes: a retrospective analysis using the FDA adverse event reporting system database. 与暴发性1型糖尿病相关的药物类别：使用FDA不良事件报告系统数据库的回顾性分析

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-01 Epub Date: 2025-01-21 DOI: 10.1080/14740338.2024.2448202

Yang Zhou, Yupeng Chen, Shan Zhang, Zhige Wen, Zifan Zhuang, Xinyao Liu, Qing Ni

{"title":"Drug classes associated with the development of fulminant type 1 diabetes: a retrospective analysis using the FDA adverse event reporting system database.","authors":"Yang Zhou, Yupeng Chen, Shan Zhang, Zhige Wen, Zifan Zhuang, Xinyao Liu, Qing Ni","doi":"10.1080/14740338.2024.2448202","DOIUrl":"10.1080/14740338.2024.2448202","url":null,"abstract":"Background: Fulminant type 1 diabetes mellitus (FT1DM) is a severe subtype of type 1 diabetes characterized by rapid onset, metabolic disturbances, and irreversible insulin secretion failure. Recent studies have suggested associations between FT1DM and certain medications, warranting further investigation.Objectives: This study aims to identify drugs associated with an increased risk of FT1DM using the FDA Adverse Event Reporting System (FAERS) database, evaluate reporting patterns, and provide actionable insights to reduce FT1DM occurrence and improve medication safety.Methods: A retrospective analysis of FAERS data from 2013 to 2023 was conducted. Drug names were standardized using text mining tools, and safety signals were evaluated using reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma Poisson Shrinker (MGPS).Results: A total of 706 FT1DM cases were identified, predominantly in older individuals and males. Nineteen drugs were implicated, including immune checkpoint inhibitors (nivolumab, ipilimumab, pembrolizumab, avelumab, durvalumab, atezolizumab), lenvatinib, eribulin, psychiatric drugs (atomoxetine, carbamazepine, lamotrigine), anti-infectives (sulfamethoxazole, trimethoprim, amoxicillin), and metabolic modulators (dapagliflozin, sitagliptin, hydrochlorothiazide, allopurinol).Conclusion: This study highlights drugs potentially triggering FT1DM and emphasizes the need for pharmacovigilance and glucose monitoring in patients treated with these medications.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"461-467"},"PeriodicalIF":3.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142964379","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Mining and analysis of adverse events associated with aducanumab: a real-world study using FDA Adverse Event Reporting System database. 挖掘和分析与aducanumab相关的不良事件：使用FDA不良事件报告系统数据库的真实世界研究。

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-01 Epub Date: 2025-01-02 DOI: 10.1080/14740338.2024.2448205

Shuangshuang Wu, Yiming Qi, Cheng Jiang, Junxian Zheng

{"title":"Mining and analysis of adverse events associated with aducanumab: a real-world study using FDA Adverse Event Reporting System database.","authors":"Shuangshuang Wu, Yiming Qi, Cheng Jiang, Junxian Zheng","doi":"10.1080/14740338.2024.2448205","DOIUrl":"10.1080/14740338.2024.2448205","url":null,"abstract":"Background: Aducanumab, a monoclonal antibody, received approval for the treatment of Alzheimer's disease in 2021. However, it remains controversial over the security of this drug. In this study, aducanumab-related adverse events (AEs) were evaluated through data mining based on the FDA Adverse Event Reporting System (FAERS) database.Research design and methods: The AE reports induced by aducanumab as the primary suspected drug were extracted from the FAERS database. The clinical characteristics of aducanumab-associated reports were analyzed. The potential new AE signals of aducanumab were explored using four disproportionality analysis methods. Furthermore, the difference in aducanumab-associated AE signals was investigated concerning sex, age, weight, dose, onset time, and continent.Results: In total, 328 reports and 793 AEs associated with aducanumab were identified. Six new AEs were identified. No significant sex and weight difference in aducanumab-related signals was found. Notably, nervous system disorders, especially 'amyloid related imaging abnormality-edema/effusion' and 'amyloid related imaging abnormality-microhaemorrhages and haemosiderin deposits,' were more frequently to be reported within 121-240 days, particularly in Europe.Conclusions: This study contributes real-world evidence regarding the safety of aducanumab.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"469-478"},"PeriodicalIF":3.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142893401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Disproportionality analysis of adverse events associated with asfotase alfa: a post-marketing study using the FDA Adverse Event Reporting System. 与asfotase alfa相关的不良事件的比例分析：使用FDA不良事件报告系统进行的上市后研究。

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-01 Epub Date: 2024-11-27 DOI: 10.1080/14740338.2024.2433566

Wang Wen, Wang Zhe, Chen Qianxiu, Sun Haixia, Fu Zongchao, Han Jing, Lv Hao

{"title":"Disproportionality analysis of adverse events associated with asfotase alfa: a post-marketing study using the FDA Adverse Event Reporting System.","authors":"Wang Wen, Wang Zhe, Chen Qianxiu, Sun Haixia, Fu Zongchao, Han Jing, Lv Hao","doi":"10.1080/14740338.2024.2433566","DOIUrl":"10.1080/14740338.2024.2433566","url":null,"abstract":"Background: Asfotase alfa (AA) is an FDA-approved enzyme replacement therapy for hypophosphatasia (HPP). Limited real-world data on its adverse events (AEs) exist. This study evaluates AA-related AEs using the U.S. FDA's Adverse Event Reporting System (FAERS) database.Methods: Reports for AA were extracted from FAERS and analyzed per FDA guidelines. AEs were categorized using MedDRA version 26.1. Disproportionality analysis was conducted using the reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian confidence propagation neural network (BCPNN), and empirical Bayes geometric mean (EBGM) algorithms. Time-to-onset (TTO) was calculated, with a Weibull shape parameter test to assess risk over time.Results: Out of 13,702,373 reports, 5,040 AA-related AEs were identified, with 198 significant preferred terms (PTs). Common AEs included injection site reactions and pain, with additional PTs for ear/labyrinth disorders and infections. The median onset for 234 AEs with reported times was 170 days (IQR 18-390 days). No significant differences in AEs were found across gender or age groups.Conclusion: Most AEs align with known data, but newly identified ear/labyrinth disorders and infections require further investigation to enhance AA's safety profile.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"435-443"},"PeriodicalIF":3.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142738912","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Safety profile of isocitrate dehydrogenase inhibitors in cancer therapy: a pharmacovigilance study of the FDA Adverse Event Reporting System. 异柠檬酸脱氢酶抑制剂在癌症治疗中的安全性：FDA不良事件报告系统的药物警戒研究。

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-01 Epub Date: 2025-01-01 DOI: 10.1080/14740338.2024.2448210

Yiming Fu, Ruxue Lv, Ruizhen Li, Wenjie Li, Zhaoze Guo

{"title":"Safety profile of isocitrate dehydrogenase inhibitors in cancer therapy: a pharmacovigilance study of the FDA Adverse Event Reporting System.","authors":"Yiming Fu, Ruxue Lv, Ruizhen Li, Wenjie Li, Zhaoze Guo","doi":"10.1080/14740338.2024.2448210","DOIUrl":"10.1080/14740338.2024.2448210","url":null,"abstract":"Background: Isocitrate dehydrogenase (IDH) inhibitors hold promise for IDH-mutated cancer patients and demonstrated favorable clinical efficacy. Nonetheless, a comprehensive understanding of the associated toxicities of IDH inhibitors remains notably lacking.Research design and methods: This pharmacovigilance analysis utilized the FDA Adverse Event Reporting System (FAERS) database to assess notable adverse events (AEs) attributed to IDH inhibitors (enasidenib and ivosidenib) from January 2018 to December 2023.Results: In the FAERS database, 2,905 enasidenib-associated and 1,289 ivosidenib-related AEs records were identified, respectively. The toxicity profiles of IDH1 and IDH2 inhibitors exhibited notable similarities. The most commonly significant system-organ classes induced by IDH inhibitors encompassed general disorders and administration site conditions, investigations, gastrointestinal disorders, and infections and infestations. The most common AEs included nausea, fatigue, diarrhea, decreased appetite, decreased platelet count, fever, pneumonia, weakness, sepsis, constipation, vomiting, rash, and reduced hemoglobin levels. Notably, temporal analysis of AEs shows enasidenib and ivosidenib have median onset times of 137 and 75 days, with distinct initial peak frequencies.Conclusion: The reversible nature of the toxicities associated with IDH inhibitors underscores their promise as a therapeutic agent with a favorable safety profile.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"479-486"},"PeriodicalIF":3.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142893402","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect? 尽管杜氏基因疗法具有免疫原性作用，但它是一种合适的治疗方法吗？

IF 3 3区医学

Expert Opinion on Drug Safety Pub Date : 2025-04-01 Epub Date: 2024-12-31 DOI: 10.1080/14740338.2024.2447072

Annie Tang, Toshifumi Yokota

{"title":"Is Duchenne gene therapy a suitable treatment despite its immunogenic class effect?","authors":"Annie Tang, Toshifumi Yokota","doi":"10.1080/14740338.2024.2447072","DOIUrl":"10.1080/14740338.2024.2447072","url":null,"abstract":"Introduction: Duchenne muscular dystrophy (DMD) is a severe X-linked disorder characterized by progressive muscle weakness and eventual death due to cardiomyopathy or respiratory complications. Currently, there is no cure for DMD, with standard treatments primarily focusing on symptom management. Using immunosuppressive measures and optimized vector designs allows for gene therapies to better address the genetic cause of the disease.Areas covered: This review evaluates the efficacy and safety of emerging DMD gene therapies as of 2024. It also discusses the potential of utrophin upregulation, gene editing, and truncated dystrophin as therapeutic strategies. It highlights safety concerns associated with these therapies, including adverse events and patient deaths. A comprehensive overview of developments covers topics such as CRISPR-Cas9 therapies, micro-dystrophin, and the potential delivery of full-length dystrophin.Expert opinion: The FDA's recent approval of delandistrogene moxeparvovec (Elevidys) underscores the promise of gene replacement therapies for DMD patients. Understanding the mechanisms behind the adverse effects and excluding patients with specific pathogenic variants may enhance the safety profiles of these therapies. CRISPR/Cas9 therapies, while promising, face significant regulatory and safety challenges that hinder their clinical application. Optimal DMD therapies should target both skeletal and cardiac muscles to be effective.","PeriodicalId":12232,"journal":{"name":"Expert Opinion on Drug Safety","volume":" ","pages":"395-411"},"PeriodicalIF":3.0,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142885302","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0