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Latest News

EHDN Platform Meeting (Webinar):
Is it time to target inflammation in Huntington’s disease?

  The Platform Meeting will take place on Friday, 30th January 2026 13.30-15.00 CET Register now   Evidence supports inflammation playing a role in Huntington’s disease pathogenesis. The mutant huntingtin protein disrupts immune cell function, centrally and peripherally. Long before symptom onset, activated microglia and astrocytes can be detected and levels of pro-inflammatory molecules like […]

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Professor Sarah Tabrizi – One of ten people who helped shape science in 2025

Congratulations to Professor Sarah Tabrizi (University College London), an investigator on the EHDN-endorsed uniQure AMT-130 gene therapy trial, on the exceptional achievement of being named one of Nature’s 10 – an annual list recognising individuals who shaped science in 2025. Read the feature here: https://www.nature.com/immersive/d41586-025-03848-1/index.html […]

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uniQure topline results: unpacking the outcome so far – update

EHDN Platform Meeting in collaboration with HDYO and EHA on uniQure topline results: Unpacking the outcomes so far The EHDN Platform Meeting took place on Tuesday, 18th November 2025. Recording is available below and on EHDN YouTube Full report summarising the meeting is available HERE Statement from EHDN Chairs on uniQure’s Topline Results Since the […]

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EHDN Platform Meeting in collaboration with HDYO and EHA – UniQure topline results: unpacking the outcome so far

  The Platform Meeting will take place on Tuesday, 18th of November, at 17:00-18:30 CET. The presentations by Prof. Anne Rosser and Prof. Sarah Tabrizi will be followed by an expert panel discussion. The event will be chaired by Prof. Åsa Petersén. All are very welcome to attend and follow this highly anticipated conversation. We […]

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Positive Topline Results for AMT-130

On 24 September, uniQure announced exciting topline results for their Phase 1/2 study of AMT-130 in HD. The high dose of AMT-130 met the study’s primary endpoint by demonstrating statistically significant slowing of disease progression (as assessed by the composite Unified Huntington’s Disease Rating Scale) at 36 months compared to a propensity score-matched external control […]

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Enroll-HD periodic dataset – PDS7

The seventh release of the Enroll-HD periodic dataset, PDS7, has been fully curated and is now available. PDS7 contains data from 30,511 Enroll-HD study participants encompassing 112,992 study visits, making it one of the largest cohort datasets available to researchers. Read more here. […]

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