Hereditary Neurological Disease Centre

11/10/2025

Join us for this webinar on why people with Huntington's disease self-neglect and what can be done to help?

11/10/2025

This event was recorded and available for your review

Next, we'll hear from Daniel Leonard regarding uniQure's current including the AMT-130 update! Listen NOW!

Watch NOW: https://fb.watch/DfcdEEHoN8/

Sign up with an address on the App for : https://digitalgrowthfirm.adalo.com/h4hdweb?target=25zsrr88t68iit4c1wckn3oin¶ms=%7B%7D

11/10/2025

11/10/2025

The HNDC offices will be closed on Tuesday, November 11th in honor of Veterans Day. We join our community in recognizing and appreciating all who have served. 🇺🇸
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For urgent assistance, please call (316) 609-3020 to connect with our live answering service.

11/05/2025

Together is how we make change. 💙
HDSA is proud to join forces with other U.S. Huntington’s disease organizations to amplify our community’s voice, especially in conversations with the FDA.
Stronger together, we’ll keep pushing forward.
(Click image to see full statement)

11/04/2025

uniQure believes that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission. This is a key shift from prior communications with the FDA in multiple Type B meetings over the past year. Consequently, the timing of the BLA submission for AMT-130 is now unclear.

11/03/2025

https://www.uniqure.com/investors-media/press-releases

Reimagining the future of healthcare through the power of gene therapy.

10/30/2025

👉 Don’t miss this! The HDSA webinar on uniQure’s AMT-130 gene therapy is 𝗵𝗮𝗽𝗽𝗲𝗻𝗶𝗻𝗴 𝘁𝗼𝗱𝗮𝘆 𝗮𝘁 𝟭 𝗣𝗠 𝗖𝗦𝗧!

The recent news from 𝘂𝗻𝗶𝗤𝘂𝗿𝗲 about their gene therapy for Huntington’s disease has sparked a lot of excitement — and questions. To help the HD community understand what this means, the Huntington’s Disease Society of America (HDSA) is hosting a special two-part webinar 𝘁𝗼𝗱𝗮𝘆!

🧬 𝗪𝗵𝗮𝘁 𝘁𝗵𝗲 𝗔𝗠𝗧-𝟭𝟯𝟬 𝗡𝗲𝘄𝘀 𝗠𝗲𝗮𝗻𝘀 𝗳𝗼𝗿 𝘁𝗵𝗲 𝗛𝗗 𝗖𝗼𝗺𝗺𝘂𝗻𝗶𝘁𝘆
📅 𝗧𝗼𝗱𝗮𝘆 – 𝗧𝗵𝘂𝗿𝘀𝗱𝗮𝘆, 𝗢𝗰𝘁𝗼𝗯𝗲𝗿 𝟯𝟬
🕐 𝟭:𝟬𝟬–𝟯:𝟬𝟬 𝗣𝗠 𝗖𝗦𝗧

𝗣𝗮𝗿𝘁 𝟭: 𝗘𝘅𝗽𝗲𝗿𝘁 𝗣𝗲𝗿𝘀𝗽𝗲𝗰𝘁𝗶𝘃𝗲𝘀
Hear from Dr. Walid Abi-Saab (uniQure) and Dr. Sarah Tabrizi (University College London) as they share insights on the latest AMT-130 results and answer key questions.

𝗣𝗮𝗿𝘁 𝟮: 𝗟𝗶𝘃𝗲 𝗤&𝗔 𝗗𝗶𝘀𝗰𝘂𝘀𝘀𝗶𝗼𝗻
Join HDSA experts for a live conversation about what this news means for HD families and what’s next.

💻 𝗝𝗼𝗶𝗻 𝘁𝗵𝗲 𝘄𝗲𝗯𝗶𝗻𝗮𝗿 𝗵𝗲𝗿𝗲: https://hdsa-org.zoom.us/.../WN_lsxS7Pq_RFeOaOwyvrNnig

Join Us for a Conversation about the uniQure AMT-130 Announcement
The recent uniQure news on gene therapy for Huntington’s disease has generated excitement and questions across the community.
📅 Thursday, October 30 | 🕑 2:00 PM ET
This event will feature:
🎥 A conversation with Dr. Walid Abi-Saab (uniQure) and Dr. Sarah Tabrizi (UCL)
💬 Followed by Q&A with HDSA's own Dr. Tam Maiuri, MaryAnn Emerick, and Allison Bartlett, Esq.
Register at https://hdsa-org.zoom.us/webinar/register/6917612418591/WN_lsxS7Pq_RFeOaOwyvrNnig to learn what the latest results mean for HD families and what to expect next.

10/28/2025

🔗 https://f.mtr.cool/drxizncoln to read more!

✨ Big news for the Huntington’s community! Recent trial results show that pridopidine may slow disease progression and preserve motor and cognitive function—especially in patients not on antidopaminergic medications.

💊 This oral therapy targets the sigma-1 receptor, helping nerve cells stay healthy and functional.

📈 The findings bring hope for a potential new disease-modifying treatment.

10/28/2025

Scientists in China developed a system to grow the specific type of brain cells that are lost in Huntington's disease, and tested it in mice. https://buff.ly/LgZxXDb