Browsing Biological Sciences by Author "Dobrenis, Kostantin"
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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
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 (2022) [Journal article]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 ...