Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for 阅读更多...
I belong to the third generation of a family affected by Huntington’s Disease, but it wasn’t until 2024, thanks to the tireless efforts of my second father, F. Cook, that we were able to confirm how the disease entered the 阅读更多...
Abu Dhabi, UAE; June 19, 2024: IROS, an Abu Dhabi-based contract research organization (part of the M42 group), has partnered with Prilenia Therapeutics, a clinical-stage biotech company, and the International Huntington’s Disease Association (IHA) to plan the first-ever clinical trial 阅读更多...
“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We believe 阅读更多...
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) protein and preserves healthy, wild-type huntingtin (wtHTT) protein for individuals 阅读更多...
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 mHTT was lowered by 21% and 43% respectively, for 5mg 阅读更多...
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 between 29 healthy volunteers and 40 participants with HD. As 阅读更多...
上个月在阿布扎比举行的中东和北非罕见病大会上，IHA 主席斯文-奥拉夫-奥尔森（Svein Olaf Olsen）就亨廷顿氏病发表了主旨演讲。他谈到了在该地区建立一个神经退行性疾病高级研究中心的迫切性，并说 阅读更多...
今天，我们亨廷顿社区非常高兴。 Uniqure公司总裁说："Uniqure宣布该公司已获得再生医学高级疗法（RMAT）"亨廷顿氏病基因疗法AMT-130研究指定"，这让我们看到了更大的希望。 阅读更多...
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氏病。她一次又一次地被误诊，因为医生拒绝考虑非裔美国人也可能遗传变异的亨廷顿舞蹈症。 阅读更多...
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