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Plasma neurofilament light, glial fibrillary acidic protein and lysosphingolipid biomarkers for pharmacodynamics and disease monitoring of GM2 and GM1 gangliosidoses patients

GM2 and GM1 gangliosidoses are genetic, neurodegenerative lysosomal sphingolipid storage disorders. The earlier the age of onset, the more severe the clinical presentation and progression, with infantile, juvenile and late-onset presentations broadly delineated into separate phenotypic subtypes. Gen...

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Detalles Bibliográficos
Autores principales:	Welford, Richard W.D., Farine, Herve, Steiner, Michel, Garzotti, Marco, Dobrenis, Kostantin, Sievers, Claudia, Strasser, Daniel S., Amraoui, Yasmina, Groenen, Peter M.A., Giugliani, Roberto, Mengel, Eugen
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2022
Materias:	Research Paper
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8856936/ https://www.ncbi.nlm.nih.gov/pubmed/35242574 http://dx.doi.org/10.1016/j.ymgmr.2022.100843

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