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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