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Therapy Trial Design in Vanishing White Matter - An Expert Consortium Opinion
April, 2022
Marjo S. van der Knaap, Joshua L. Bonkowsky, Adeline Vanderver, Raphael Schiffmann, Ingeborg Krägeloh-Mann, Enrico Bertini, Genevieve Bernard, Seyed Ali Fatemi, Nicole I. Wolf, Elise Saunier-Vivar, Robert Rauner, Hanka Dekker, Pieter van Bokhoven, Peter van de Ven, Prisca S. Leferink
Neurol Genet Apr 2022, 8 (2) e657; DOI: 10.1212/NXG.0000000000000657
Natural History of Vanishing White Matter
August 2018
Hamilton EMC, van der Lei HDW, Vermeulen G, Gerver JAM, Lourenço CM, Naidu S, Mierzewska H, Gemke RJBJ, de Vet HCW, Uitdehaag BMJ, Lissenberg-Witte BI; VWM Research Group, van der Knaap MS
Ann Neurol; 84(2):274-288. doi: 10.1002/ana.25287. Epub 2018 Sep 6. PMID: 30014503; PMCID: PMC6175238.