A trial of topiramate for patients with hereditary spinocerebellar ataxia. Issue 2 (26th February 2023)

• Record Type: Journal Article
• Authors: Miura, Shiroh; Sawada, Ryusuke; Yorita, Akiko; Kida, Hiroshi; Kamada, Takashi; Yamanishi, Yoshihiro
• Is Part Of: Clinical case reports. Volume 11:Issue 2(2023)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2023-02-26
• Subjects: drug repositioning -- spinocerebellar ataxia -- topiramate; Medicine -- Periodicals; 616.09
• DOI: 10.1002/ccr3.6980
• Languages: English
• ISSNs: 2050-0904

Abstract

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. Abstract : We proposed a computational drug repositioning approach, performed a screening of exsiting drugs, and selected topiramate as a drug candidate for spinocerebellar ataxia (SCA). This pilot study suggests that topiramate could be effective for some patients with hereditary SCA.