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托吡酯用于遗传性脊髓小脑共济失调患者的试验。

A trial of topiramate for patients with hereditary spinocerebellar ataxia.

作者信息

Miura Shiroh, Sawada Ryusuke, Yorita Akiko, Kida Hiroshi, Kamada Takashi, Yamanishi Yoshihiro

机构信息

Department of Neurology and Geriatric Medicine Ehime University Graduate School of Medicine Toon Ehime Japan.

Department of Pharmacology Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences Kita-ku Okayama Japan.

出版信息

Clin Case Rep. 2023 Feb 26;11(2):e6980. doi: 10.1002/ccr3.6980. eCollection 2023 Feb.

DOI:10.1002/ccr3.6980
PMID:36855409
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9968455/
Abstract

In an open pilot trial, six patients with various hereditary forms of spinocerebellar ataxia (SCA) were assigned to topiramate (50 mg/day) for 24 weeks. Four patients completed the protocol without adverse events. Of these four patients, topiramate was effective for three patients. Some patients with SCA could respond to treatment with topiramate.

摘要

在一项开放性试验中,6例患有各种遗传性脊髓小脑共济失调(SCA)的患者被分配接受托吡酯治疗(50毫克/天),为期24周。4例患者完成了方案且无不良事件发生。在这4例患者中,托吡酯对3例患者有效。一些SCA患者可能对托吡酯治疗有反应。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/813b/9968455/f898b4d690d8/CCR3-11-e6980-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/813b/9968455/f898b4d690d8/CCR3-11-e6980-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/813b/9968455/f898b4d690d8/CCR3-11-e6980-g002.jpg

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本文引用的文献

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Episodic Vestibulocerebellar Ataxia Associated with a Missense Variant.与错义变异相关的发作性前庭小脑共济失调
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Spinocerebellar ataxia 27 with a novel nonsense variant (Lys177X) in FGF14.伴有成纤维细胞生长因子14(FGF14)中新型无义变异(Lys177X)的脊髓小脑共济失调27型
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