Zoeken
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
August, 2023
Daphne H. Schoenmakers, Prisca S. Leferink, Adeline Vanderver, Joshua L. Bonkowsky, Ingeborg Krägeloh-Mann, Geneviève Bernard, Enrico Bertini, Ali Fatemi, Brent L. Fogel, Nicole I. Wolf, Donna Skwirut, Allyson Buck, Brett Holberg, Elise F. Saunier-Vivar, Robert Rauner, Hanka Dekker, Pieter van Bokhoven, Menno D. Stellingwerff, Johannes Berkhof & Marjo S. van der Knaap
BMC Neurol 23, 305 (2023). https://doi.org/10.1186/s12883-023-03354-9
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
Childhood Ataxia with Central Nervous System Hypomyelination / Vanishing White Matter
February 2003
van der Knaap MS, Fogli A, Boespflug-Tanguy O, Abbink TEM, Schiffmann R.
GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2020. PMID: 20301435 Bookshelf ID: NBK1258
Leukoencephalopathy with vanishing white matter: a review
October, 2010
Bugiani M, Boor I, Powers JM, Scheper GC, van der Knaap MS
J Neuropathol Exp Neurol; 69(10):987-96. doi: 10.1097/NEN.0b013e3181f2eafa. PMID: 20838246.
Vanishing white matter disease
June 29, 2021
van der Knaap MS, Pronk JC, Scheper GC
Lancet Neurol. 2006 May;5(5):413-23. doi: 10.1016/S1474-4422(06)70440-9. PMID: 16632312.