Visual evoked potentials of Niemann-Pick type C1 mice reveal an impairment of the visual pathway tha...
Visual evoked potentials of Niemann-Pick type C1 mice reveal an impairment of the visual pathway that is rescued by 2-hydroxypropyl-ß-cyclodextrin
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England: BioMed Central Ltd
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English
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England: BioMed Central Ltd
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The lysosomal storage disorder, Niemann Pick type C1 (NPC1), presents a variable phenotype including neurovisceral and neurological symptoms. 2-Hydroxypropyl-ß-cyclodextrin (HPßCD)-based therapies are presently the most promising route of intervention. While severe cerebellar dysfunction remains the main disabling feature of NPC1, sensory functions...
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Visual evoked potentials of Niemann-Pick type C1 mice reveal an impairment of the visual pathway that is rescued by 2-hydroxypropyl-ß-cyclodextrin
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4603821
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4603821
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ISSN
1750-1172
E-ISSN
1750-1172
DOI
10.1186/s13023-015-0348-0