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 by Schoenmakers, Daphne H, Leferink, Prisca S, Vanderver, Adeline, Bonkowsky, Joshua L, Krägeloh-Mann, Ingeborg, Bernard, Geneviève, Bertini, Enrico, Fatemi, Ali, Fogel, Brent L, Wolf, Nicole I, Skwirut, Donna, Buck, Allyson, Holberg, Brett, Saunier-Vivar, Elise F, Rauner, Robert, Dekker, Hanka, van Bokhoven, Pieter, Stellingwerff, Menno D, Berkhof, Johannes, van der Knaap, Marjo S

Get full text