Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and patient advocates.

Autor: Schoenmakers, Daphne H.1,2,3 (AUTHOR), Leferink, Prisca S.4 (AUTHOR), Vanderver, Adeline5,6 (AUTHOR), Bonkowsky, Joshua L.7,8 (AUTHOR), Krägeloh-Mann, Ingeborg9 (AUTHOR), Bernard, Geneviève10,11 (AUTHOR), Bertini, Enrico12 (AUTHOR), Fatemi, Ali13 (AUTHOR), Fogel, Brent L.14 (AUTHOR), Wolf, Nicole I.1,2 (AUTHOR), Skwirut, Donna15,16 (AUTHOR), Buck, Allyson16 (AUTHOR), Holberg, Brett16 (AUTHOR), Saunier-Vivar, Elise F.17 (AUTHOR), Rauner, Robert15 (AUTHOR), Dekker, Hanka18 (AUTHOR), van Bokhoven, Pieter4 (AUTHOR), Stellingwerff, Menno D.1,2 (AUTHOR), Berkhof, Johannes19 (AUTHOR), van der Knaap, Marjo S.1,2,20 (AUTHOR) ms.vanderknaap@amsterdamumc.nl
Zdroj: BMC Neurology. 8/17/2023, Vol. 23 Issue 1, p1-15. 15p.
Databáze: Academic Search Ultimate
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