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

por 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.
Publicado 2023