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MDS-PAS and EHDN Joint Online Course Series: Multidisciplinary Management of Huntington’s Disease

Serie de Cursos Conjuntos en Línea MDS-PAS y EHDN: Manejo Multi- disciplinario de la Enfermedad de Huntington Course Format Start Date End Date Live Online Fri, Aug 15, 2025 Fri, Aug 22, 2025 Program Description Serie de Cursos Conjuntos en Línea MDS-PAS y EHDN: Manejo Multi- disciplinario de la Enfermedad de Huntington: Retos actuales y […]

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PIVOT-HD Achieves Primary Endpoint

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 […]

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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 […]

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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 […]

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