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

BACKGROUND: The leukodystrophy “Vanishing White Matter” (VWM) is an orphan disease with neurological decline and high mortality. Currently, VWM has no approved treatments, but advances in understanding pathophysiology have led to identification of promising therapies. Several investigational medicin...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Study Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10433679/
https://www.ncbi.nlm.nih.gov/pubmed/37592248
http://dx.doi.org/10.1186/s12883-023-03354-9

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