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PTC Therapeutics 与诺华公司就 PTC518 亨廷顿氏症项目达成全球许可与合作协议

December 2, 2024 – PTC to receive $1.0B in cash at closing –– PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones –– PTC to share profits in the U.S. and […]

Sage Therapeutics 停止开发用于治疗 HD 的 dalzanemdor

The Phase 2 DIMENSION Study did not meet its primary endpoint Dalzanemdor was generally well-tolerated; no new safety signals were observed Based on these data, the Company does not plan […]

伙伴关系的力量。瑞士和中国树立了榜样。

In this video, Professor Jean-Marc Burgunder, head of the Huntington’s Disease (HD) centre in Bern Switzerland, and Xi Cao, President of the Chinese HD Association talk about the benefits of […]

宜居生活》--一本重点介绍世界各地受 HD 影响的人们的故事的新书

PRESS RELEASE – October 25, 2024, Moscow, Idaho, USAWe are pleased to announce the launch of a new book about Huntington’s disease (HD) called, Livable Lives: Conversations with the Huntington’s […]

LoQus23 Therapeutics 公司宣布融资 3500 万英镑，用于开发一种抑制亨廷顿氏病体细胞扩增的新药

Cambridge, UK, 2 October 2024 – LoQus23 Therapeutics Ltd (“LoQus23”), a private biotechnology company investigating small molecule drugs that could stop DNA instability and slow neurodegeneration in Huntington’s Disease, myotonic dystrophy […]

中东和北非罕见病大会

人类发展青年联盟大会

FDA 授予 PTC518 亨廷顿氏病项目快速通道称号

PTC518 is an oral medication that reduces the production of the mutated Huntingtin protein that causes disease progression.   Programs with Fast Track designation can benefit from early interactions with the FDA, and may be […]

Prilenia 公司治疗亨廷顿氏症的普利多匹定接受欧洲上市许可审查

Prilenia 是一家处于临床阶段的生物技术公司，致力于开发新型疗法以延缓神经退行性疾病和神经发育障碍的进展。

不懈探索纪事：创建基金会

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 […]