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PTC518 PIVOT-HD Study Achieves Primary Endpoint

May 5, 2025 PDF Version – Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 – – Favorable dose-dependent trends across clinical scales in Stage 2 […]

UniQure оголошує про надання препарату AMT-130 статусу проривної терапії для лікування хвороби Хантінгтона від Управління з контролю за продуктами і ліками США

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~ ~ Additional regulatory update and guidance on the Biologics License Application […]

Погляд назад на Конгрес HDYO 2025

The HDYO Congress 2025 brought together young people, families, researchers, and professionals from around the world for an unforgettable event dedicated to the Huntington’s Disease community. Over three inspiring days, […]

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