Association of NOTCH3 Variant Risk Category With 2-Year Clinical and Radiologic Small Vessel Disease Progression in Patients With CADASIL.

Minne N CerfontaineRemco J HackBenno Gesierich Marco DueringMarie-Noëlle W Witjes-AnéMar Rodríguez-GirondoGido GravesteijnJulie RuttenSaskia A J Lesnik Oberstein

Published in: Neurology (2024)

variants. This has important implications for clinical trial design and disease prediction and monitoring in the clinic. Moreover, we show that MSMD is a promising outcome measure for trials enrolling premanifest individuals.

Keyphrases

clinical trial
primary care
copy number
cell proliferation
phase ii
study protocol
double blind
open label
randomized controlled trial
gene expression
phase iii