On 5 May 2025, PTC Therapeutics shared a press release reporting results from the PIVOT-HD study of PTC518 (votoplam), a small molecule splicing modifier. This Phase 2, 12-month trial assessed the pharmacodynamic effect and safety of PTC518 at 5 mg and 10 mg compared to placebo in participants defined as HD-ISS Stage 2 or Stage […]
Kategoria: Uncategorized
FDA Breakthrough Therapy Designation for AMT-130
uniQure announced on 17 April 2025 that the FDA has granted Breakthrough Therapy designation for AMT-130 gene therapy in the treatment of HD, meaning that development and review will be expedited. This designation is supported by clinical data from ongoing Phase I/II trials, including interim data announced in July 2024 showing dose-dependent slowing of disease […]
GENERATION HD2 to continue
On 17 April 2025, Roche released a community letter announcing that GENERATION HD2 will continue following planned interim analysis by the independent Data Monitoring Committee. Enrollment has completed for this randomised, double-blind study of tominersen, but participants on the 60 mg dose will move to the higher dose (100 mg every 16 weeks), given the […]
Deep Brain Stimulation against Chorea in HD: Results and Reflections
The recording is now available on YouTube. […]
In Memoriam: Tim McLean
It is with very heavy hearts that we share the sad news that Tim McLean, our wonderful colleague and dear friend, passed away on 31 March 2025. […]
EHDN Newsletter – 54th edition
The EHDN newsletter (archive) aims to communicate the network’s activities and other developments of interest in the field of Huntington’s disease to the lay community, healthcare professionals and scientists. It appears three times a year (March 1, July 1, Nov 1). To receive EHDN news going forward, please subscribe to the EHDN news at the […]
Next deadline for EHDN seed funds: March 1st 2025
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HDYO International Young Adults Congress
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EHDN & Enroll-HD 2024 Report, prepared by Catherine Deeprose
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EHDN Imaging Working Group (iWG) newsletter
The EHDN Imaging Working Group is now producing a regular newsletter. The newsletter provides relevant updates on imaging studies, research advancements, and innovative techniques in neuroimaging. Welcome to the inaugural edition of the Imaging Working Group (iWG) NEWSLETTER Edition 1 | December 2024 […]