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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next steps for Pridopidine

On 25 July, Prilenia shared that the application for pridopidine had been rejected for marketing authorisation in HD by the European Medicines Agency’s Committee for Medicinal Products for Human Use. The application was based on post-hoc analyses from the EHDN-endorsed, Phase 3 PROOF-HD study, suggesting that the orally administered sigma-1 receptor agonist may offer potential […]

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