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Latest News

Clinical Trial Update: uniQure alignment with FDA on path forward for AMT-130 in HD

Clinical Trial Update: On 10th December, uniQure shared the exciting news about alignment with the US Food and Drug Administration (FDA) on a path forward for AMT-130 gene therapy in Huntington’s disease. The FDA agreed that the basis for a Biologics License Application for Accelerated Approval of AMT-130 may include: 1. Data from ongoing phase […]

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EHDN/MDS (ES) Fellowship programme now open for applications

The main objective of the fellowship programme is to motivate and facilitate the training of young professionals (neurologists, psychiatrists, psychologists, physiotherapists, clinical geneticists or other healthcare professionals) working in HD clinical practice through the sharing of knowledge and experience. For more information please read the fellowship programme page. […]

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Clinical Trial Update: Results from the Phase 2 DIMENSION Study

Sage reported on 20th November that the EHDN-endorsed 12-week placebo-controlled, Phase 2 DIMENSION study of Sage-718 (dalzanemdor) has failed to meet its primary or secondary endpoints. Given these results, the dalzanemdor programme will close, including the currently ongoing open-label extension study PURVIEW. While these are disappointing developments, we recognise and are grateful for the progress […]

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Prizes awarded to Factor-H

Congratulations to Factor-H for the hugely well-deserved recognition of their work from Amgen (2024 Amgen/MIT Solve award) and MDS (Public Service Award). Read more here: Factor-H is Awarded the 2024 Amgen Prize! and Factor-H President receives the 2024 Public Service Award from the MDS. […]

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Alnylam Pharmaceuticals Global study endorsed* by EHDN

Study aims and goal: ALN-HTT02 is a novel C16-siRNA conjugate for huntingtin-lowering. This Phase Ib study aims to evaluate ALN-HTT02 in adults with Huntington’s Disease. No. of study participants and sites: The study plans to enrol participants from ~20 sites in Canada, Germany, UK and US. For further information see the clinical trial registration. *EHDN […]

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