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首页> 外文期刊>Epilepsy & behavior: E&B >Individualized treatment approaches: Fenfluramine, a novel antiepileptic medication for the treatment of seizures in Dravet syndrome
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Individualized treatment approaches: Fenfluramine, a novel antiepileptic medication for the treatment of seizures in Dravet syndrome

机译：个性化治疗方法：Fenfluramine，一种新型抗癫痫药物，用于治疗Dravet综合征癫痫发作

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摘要

Dravet syndrome is a rare and severe encephalopathy that first presents in infancy with seizures refractory to conventional antiepileptic drugs. Forty-five percent of patients report four or more tonic-clonic seizures per month despite multidrug regimens. Fenfluramine, an amphetamine derivative, was initially developed as an appetite suppressant with a serotonergic mechanism of action. Clinical observation of a potential antiepileptic activity in a small homogeneous cohort of patients combined with a genetic workup of these patients led to the hypothesis of fenfluramine as a treatment for seizures in Dravet syndrome. This concept was successfully evaluated in a zebrafish model and led to a Phase 3 trial of fenfluramine to treat seizures in children with Dravet syndrome. Preliminary results of the trial suggest that fenfluramine may be a highly effective, well-tolerated treatment for patients with Dravet syndrome. This short review summarizes the history of use of fenfluramine from the initial clinical observations followed by preclinical studies and subsequent successful clinical trial.
机译：Dravet综合征是一种罕见和严重的脑病，首先用癫痫发作到常规的抗癫痫药物难以发病。尽管多药中规范，但四十五名患者每月报告四种或更多种补品克隆癫痫发作。芬氟胺，一种安非他明衍生物，最初被开发为具有血清致命作用机制的食欲抑制剂。临床观察小型均匀群体中潜在的抗癫痫活性，结合这些患者的遗传余量导致Fenfluramine的假设作为Dravet综合征癫痫发作的治疗。该概念在斑马鱼模型中成功评估，并导致芬氟胺的第3期试验，以治疗Dravet综合征的儿童癫痫发作。试验的初步结果表明，芬氟胺对于Dravet综合征患者来说可能是一种非常有效的，耐受性良好的治疗方法。此简短评论总结了初期临床观察中芬氟胺的使用史，随后是临床前研究和随后的成功临床试验。

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