Department of Pharmaceutics, School of Pharmaceutical Sciences, Delhi Pharmaceutical Sciences and Research University (DPSRU), New Delhi 110017, India.
Department of Pharmaceutics, College of Pharmacy, Jazan University, Jazan, P. Box No. 114, Saudi Arabia.
Curr Pharm Des. 2024;30(19):1472-1489. doi: 10.2174/0113816128288707240404051856.
Friedreich's Ataxia (FRDA) is a rare hereditary neurodegenerative disorder characterized by progressive ataxia, cardiomyopathy, and diabetes. The disease is caused by a deficiency of frataxin, a mitochondrial protein involved in iron-sulfur cluster synthesis and iron metabolism.
This review aims to summarize recent advances in the development of treatment strategies for FRDA, with a focus on potential drug candidates and their mechanisms of action.
A comprehensive literature search was conducted using various authentic scientific databases to identify studies published in the last decade that investigated potential treatment strategies for FRDA. The search terms used included "Friedreich's ataxia", "treatment", "drug candidates", and "mechanisms of action".
To date, only one drug got approval from US-FDA in the year 2023; however, significant developments were achieved in FRDA-related research focusing on diverse therapeutic interventions that could potentially alleviate the symptoms of this disease. Several promising drug candidates have been identified for the treatment of FRDA, which target various aspects of frataxin deficiency and aim to restore frataxin levels, reduce oxidative stress, and improve mitochondrial function. Clinical trials have shown varying degrees of success, with some drugs demonstrating significant improvements in neurological function and quality of life in FRDA patients.
While there has been significant progress in the development of treatment strategies for FRDA, further research is needed to optimize these approaches and identify the most effective and safe treatment options for patients. The integration of multiple therapeutic strategies may be necessary to achieve the best outcomes in FRDA management.
弗里德赖希共济失调（FRDA）是一种罕见的遗传性神经退行性疾病，其特征为进行性共济失调、心肌病和糖尿病。该病由 frataxin 缺乏引起，frataxin 是一种参与铁硫簇合成和铁代谢的线粒体蛋白。
本综述旨在总结 FRDA 治疗策略的最新进展，重点关注潜在的药物候选物及其作用机制。
使用各种真实科学数据库进行全面的文献检索，以确定过去十年中研究 FRDA 潜在治疗策略的研究。使用的搜索词包括“Friedreich's ataxia”、“treatment”、“drug candidates”和“mechanisms of action”。
迄今为止，只有一种药物于 2023 年获得美国食品药品监督管理局（FDA）批准；然而，FRDA 相关研究在聚焦于多种治疗干预措施方面取得了显著进展，这些干预措施可能潜在地缓解这种疾病的症状。已经确定了几种有前途的药物候选物用于 FRDA 的治疗，这些药物针对 frataxin 缺乏的各个方面，旨在恢复 frataxin 水平、减轻氧化应激和改善线粒体功能。临床试验取得了不同程度的成功，一些药物在 FRDA 患者的神经功能和生活质量方面显示出显著改善。
尽管 FRDA 治疗策略的开发取得了重大进展，但仍需要进一步研究来优化这些方法，并为患者确定最有效和安全的治疗选择。可能需要整合多种治疗策略以实现 FRDA 管理的最佳结果。
