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Paper Details

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.
Lancet
225
2017
2-hydroxypropyl-β-cyclodextrin, 2-hydroxypropyl-β-cyclodextrins, 24(S)-HC, 24(S)-hydroxycholesterol, 24[S]-HC, CSF, CSF protein, HPβCD, Mid-frequency to high-frequency hearing loss, NPC, NPC Disease, NPC1, Niemann-Pick Disease, Niemann-Pick disease, type C1, cerebellar Purkinje cell, cholesterol, hepatocellular carcinoma, lysosomal storage disorder, mastoiditis, mouse, neurodegeneration, neurological, neuronal pathology, βCD
Author NameAffiliation
Kelly A KingNational Institute on Deafness and Other Communication Disorders, National Institutes of Health
Lisa A Weissfeld
Ariane SoldatosNational Institute of Neurological Disorders and Stroke, National Institutes of Health
Kimberly A Walters
William J PavanNational Human Genome Research Institute, National Institutes of Health
William J PavanNational Human Genome Research Institute, National Institutes of Health
Carmen C BrewerNational Institute on Deafness and Other Communication Disorders, National Institutes of Health
Christopher P AustinNational Center for Advancing Translational Sciences, National Institutes of Health
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Release 1.2