{"title":"亨廷顿氏病临床试验更新：2024年9月。","authors":"Mena Farag, Sarah J Tabrizi, Edward J Wild","doi":"10.1177/18796397241293955","DOIUrl":null,"url":null,"abstract":"<p><p>In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent study. We also discuss 24-month interim results from uniQure's AMT-130 program and 28-week follow-up results from Wave Life Sciences' SELECT-HD clinical trial of WVE-003. Additionally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.</p>","PeriodicalId":16042,"journal":{"name":"Journal of Huntington's disease","volume":"13 4","pages":"409-418"},"PeriodicalIF":2.1000,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Huntington's Disease Clinical Trials Update: September 2024.\",\"authors\":\"Mena Farag, Sarah J Tabrizi, Edward J Wild\",\"doi\":\"10.1177/18796397241293955\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent study. We also discuss 24-month interim results from uniQure's AMT-130 program and 28-week follow-up results from Wave Life Sciences' SELECT-HD clinical trial of WVE-003. Additionally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.</p>\",\"PeriodicalId\":16042,\"journal\":{\"name\":\"Journal of Huntington's disease\",\"volume\":\"13 4\",\"pages\":\"409-418\"},\"PeriodicalIF\":2.1000,\"publicationDate\":\"2024-11-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Huntington's disease\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/18796397241293955\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/10/26 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"NEUROSCIENCES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Huntington's disease","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/18796397241293955","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/10/26 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"NEUROSCIENCES","Score":null,"Total":0}
引用次数: 0
摘要
在本期的亨廷顿病临床试验更新中，我们扩展了PTC Therapeutics正在进行的PTC518扩展研究，包括来自母研究的12个月中期结果。我们还将讨论unique的AMT-130项目的24个月中期结果，以及Wave Life Sciences的SELECT-HD临床试验WVE-003的28周随访结果。此外，我们还提供所有目前已注册和正在进行的亨廷顿病临床试验的综合清单。
Huntington's Disease Clinical Trials Update: September 2024.
In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent study. We also discuss 24-month interim results from uniQure's AMT-130 program and 28-week follow-up results from Wave Life Sciences' SELECT-HD clinical trial of WVE-003. Additionally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.
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