Therapy Trial Design in Vanishing White Matter: An Expert Consortium Opinion

Vanishing white matter (VWM) is a leukodystrophy caused by recessive variants in the genes EIF2B1-EIF2B5. It is characterized by chronic neurologic deterioration with superimposed stress-provoked episodes of rapid decline. Disease onset spans from the antenatal period through senescence. Age at onse...

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Detalles Bibliográficos
Autores principales: van der Knaap, Marjo S., Bonkowsky, Joshua L., Vanderver, Adeline, Schiffmann, Raphael, Krägeloh-Mann, Ingeborg, Bertini, Enrico, Bernard, Genevieve, Fatemi, Seyed Ali, Wolf, Nicole I., Saunier-Vivar, Elise, Rauner, Robert, Dekker, Hanka, van Bokhoven, Pieter, van de Ven, Peter, Leferink, Prisca S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Wolters Kluwer 2022
Materias:
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Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8811717/
https://www.ncbi.nlm.nih.gov/pubmed/35128050
http://dx.doi.org/10.1212/NXG.0000000000000657

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8811717/
https://www.ncbi.nlm.nih.gov/pubmed/35128050
http://dx.doi.org/10.1212/NXG.0000000000000657

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