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Ditte Nedergaard Mikkelsen on Muck Rack

Ditte Nedergaard Mikkelsen

As seen in: Nature
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Articles

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

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By Daniel Dumas, Imogen Swift, Jesminne Castricum, Anders Riisager, Nina Haagenrud Schultz, Paul Little, Ove Pedersen, Eva Thijssen, Anders Nykjaer, Philip Kremer, Mads Kjolby, Louise Klem, Sofie L. Frandsen, Ditte Nedergaard Mikkelsen, Laerke Fensbaek, Yalçin Yavuz, Okke Merton, Simone Prichardt, Graham M. Wynne
| Nature Verified
Abstract GRN haploinsufficiency reduces progranulin and causes frontotemporal dementia (FTD-GRN). VES001, an oral small-molecule sortilin inhibitor, increases progranulin without downregulating sortilin. Preclinically, VES001 bound sortilin selectively and elevated progranulin in plasma, cerebrospinal fluid (CSF), and hippocampal interstitial fluid of male rats.
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