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Hoffmann-La Roche POINT-HD study endorsed* by EHDN

Study aims and goal: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RG6496 in Huntington’s Disease. No. of study participants and sites: The study plans to enrol participants from 2 sites in Australia and New Zealand. For further information see the clinical trial registration. *EHDN endorsement: The EHDN endorses protocols it finds […]

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

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 IL-6 and TNF-α are found increased in CSF and plasma. Far from being just a byproduct of […]

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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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