Preclinical and clinical investigation of VES001, an oral small molecule sortilin receptor inhibitor for the treatment of frontotemporal dementia: a first-in-human randomized placebo-controlled phase 1 trial.
Nature communications · 2026
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HumansMaleSortilinFemaleAdaptor Proteins, Vesicular TransportAdultDouble-Blind MethodAnimalsFrontotemporal DementiaAdministration, OralProgranulinsRatsYoung AdultMiddle Aged
Come citare questo studio
Louise Klem, Daniel Dumas, Imogen J Swift, Sofie L Frandsen, Ditte Nedergaard Mikkelsen, Laerke Fensbaek, Yalçin Yavuz, Okke Merton, Jesminne Castricum, Simone Prichardt, Graham M Wynne, Anders Riisager, Nina Schultz, Paul B Little, Ove Pedersen, Eva Thijssen, Anders Nykjaer, Philip Kremer, Mads Kjolby. (2026). Preclinical and clinical investigation of VES001, an oral small molecule sortilin receptor inhibitor for the treatment of frontotemporal dementia: a first-in-human randomized placebo-controlled phase 1 trial.. Nature communications. DOI: 10.1038/s41467-026-77638-2
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