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

Since the release of uniQure’s topline results from the AMT-130 gene therapy trial — followed by further updates in recent weeks — there has been widespread interest across the Huntington’s disease community. This EHDN Platform Meeting, organised in collaboration with HDYO and EHA, will bring together leading experts and advocates to unpack what the results tell us so far and what they may mean for the field — scientifically, clinically, and for families impacted by HD.
Presentations by Prof. Anne Rosser and Prof. Sarah Tabrizi will be followed by a panel discussion chaired by Prof. Åsa Petersén, offering scientific and community perspectives on the recent results and reflecting on the current state of play.

Link to the AMT-130 study (https://clinicaltrials.gov/study/NCT05243017) Press release from uniQure (dated 24 September 2025) uniQure Announces Positive Topline Results from Pivotal Phase I/II Study of AMT-130 in Patients with Huntington’s Disease Press release from uniQure (regulatory update from 3 November 2025) uniQure Provides Regulatory Update on AMT-130 for Huntington’s Disease

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