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Wave 生命科学公司宣布 SELECT-HD 1b/2a 期试验取得积极成果，首次在临床上证明可降低亨廷顿氏症患者的等位基因选择性突变亨廷廷蛋白水平

Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT) […]

PTC 今天宣布，其口服 PTC518 的 2 期 PIVOT-HD 研究在 12 个月内取得了令人鼓舞的结果  

At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where […]

Sage Therapeutics 公司的 2 期研究证实了亨廷顿氏症对认知的影响

Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease.  Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference […]

中东和北非的最新情况

IHA President Svein Olaf Olsen was keynote speaker on Huntington’s Disease at the MENA Congress for Rare Diseases in Abu Dhabi last month. He spoke of the urgent need to […]

FDA 授予 uniQure 再生医学先进疗法 (RMAT) 称号

Today we are very happy in the Huntington community.  The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130 […]

我们存在塔尼塔-艾伦讲述了她为在美国获得基因检测而进行的斗争

塔尼塔-艾伦在她的回忆录《我们存在》中讲述了她很难将自己的症状正确诊断为亨廷顿舞蹈症。她一次又一次地被误诊，因为医生拒绝[......］

高清患者免费在线心理治疗计划

Doctors at the University of Reading (UK) have developed a free online psychotherapy programme for people with Huntington’s Disease.  It’s part of a research project that looks at how people […]

巴基斯坦资助成立亨廷顿氏病新协会 - HDSOP

Founding of the Huntington’s Disease Society of Pakistan April 24th 2024 Last week another national association was created.  The Huntington’s Disease Society of Pakistan (HDSOP) joined the growing  list of […]

SOM 生物技术公司完成了治疗亨廷顿舞蹈症的 IIb 期研究的招募过程

SOM生物技术公司（SOM Biotech）是一家基于独特的专有人工智能平台（SOMAIPRO®）的临床阶段药物发现和开发公司，该公司欣喜地宣布，IIb期临床 [...] 的招募工作已经完成。

Prilenia 计划向欧盟提交普利多匹定治疗亨廷顿氏症的上市许可申请

Prilenia 公司今天宣布，他们计划向欧盟提交普利多匹定治疗亨廷顿氏症的上市许可申请。据迈克尔-[...]博士称