{"id":"https://openalex.org/W2802807799","doi":"https://doi.org/10.5772/intechopen.71970","title":"Use of 2-Hydroxypropyl-Beta-Cyclodextrin for Niemann-Pick Type C Disease","display_name":"Use of 2-Hydroxypropyl-Beta-Cyclodextrin for Niemann-Pick Type C Disease","publication_year":2018,"publication_date":"2018-04-18","ids":{"openalex":"https://openalex.org/W2802807799","doi":"https://doi.org/10.5772/intechopen.71970","mag":"2802807799"},"language":"en","primary_location":{"id":"doi:10.5772/intechopen.71970","doi":"https://doi.org/10.5772/intechopen.71970","is_oa":true,"landing_page_url":"https://doi.org/10.5772/intechopen.71970","pdf_url":"https://www.intechopen.com/citation-pdf-url/57792","source":{"id":"https://openalex.org/S4306463447","display_name":"InTech eBooks","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"ebook platform"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Cyclodextrin - A Versatile Ingredient","raw_type":"book-chapter"},"volume":{"id":"https://openalex.org/W2888200482","display_name":"Cyclodextrin - A Versatile Ingredient"},"type":"book-chapter","is_traditional":true,"indexed_in":["crossref"],"open_access":{"is_oa":true,"oa_status":"hybrid","oa_url":"https://www.intechopen.com/citation-pdf-url/57792","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5021641803","display_name":"Juan Eduardo Meg\u00edas\u2010Vericat","orcid":"https://orcid.org/0000-0002-0369-5341","observed_orcids":["https://orcid.org/0000-0002-0369-5341"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Juan Eduardo Meg\u00edas-Vericat","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"middle","author":{"id":"https://openalex.org/A5012084751","display_name":"Ana Alejandra Garc\u00eda-Robles","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Ana Alejandra Garc\u00eda-Robles","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]},{"author_position":"last","author":{"id":"https://openalex.org/A5089930932","display_name":"Jos\u00e9 Lu\u00eds Poveda","orcid":"https://orcid.org/0000-0003-1800-6198","observed_orcids":["https://orcid.org/0000-0003-1800-6198"]},"institutions":[],"countries":[],"is_corresponding":false,"raw_author_name":"Jos\u00e9 Luis Poveda","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]}],"institutions":[],"countries_distinct_count":0,"institutions_distinct_count":0,"corresponding_author_ids":[],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":0.9964,"has_fulltext":true,"cited_by_count":4,"citation_normalized_percentile":{"value":0.77338129,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":87,"max":93},"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":1.0,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},"topics":[{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":1.0,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}},{"id":"https://openalex.org/T12604","display_name":"Glycogen Storage Diseases and Myoclonus","score":0.0,"subfield":{"id":"https://openalex.org/subfields/1303","display_name":"Biochemistry"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T14415","display_name":"Biomedical Research and Pathophysiology","score":0.0,"subfield":{"id":"https://openalex.org/subfields/2727","display_name":"Nephrology"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/niemann-pick-disease-type-c","display_name":"Niemann-Pick disease type C","score":0.9729999899864197},{"id":"https://openalex.org/keywords/hydroxypropyl-beta-cyclodextrin","display_name":"hydroxypropyl-\u03b2-cyclodextrin","score":0.9779999852180481},{"id":"https://openalex.org/keywords/intrathecal-drug-delivery","display_name":"intrathecal drug delivery","score":0.6890000104904175},{"id":"https://openalex.org/keywords/lysosomal-storage-disorders","display_name":"lysosomal storage disease","score":0.8830000162124634},{"id":"https://openalex.org/keywords/cholesterol-homeostasis","display_name":"cholesterol homeostasis","score":0.8640000224113464},{"id":"https://openalex.org/keywords/disease-progression","display_name":"disease progression","score":0.640999972820282},{"id":"https://openalex.org/keywords/blood-brain-barrier-permeability","display_name":"blood-brain barrier permeability","score":0.7260000109672546},{"id":"https://openalex.org/keywords/hearing-loss","display_name":"hearing loss","score":0.6320000290870667},{"id":"https://openalex.org/keywords/drug-toxicity","display_name":"drug toxicity","score":0.6919999718666077},{"id":"https://openalex.org/keywords/neurodegeneration","display_name":"neurodegeneration","score":0.5669999718666077}],"concepts":[{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.6213800311088562},{"id":"https://openalex.org/C197934379","wikidata":"https://www.wikidata.org/wiki/Q2047938","display_name":"Adverse effect","level":2,"score":0.5918692946434021},{"id":"https://openalex.org/C2780635549","wikidata":"https://www.wikidata.org/wiki/Q2067267","display_name":"Niemann\u2013Pick disease, type C","level":3,"score":0.4932188391685486},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.4654788374900818},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.45220163464546204},{"id":"https://openalex.org/C157820659","wikidata":"https://www.wikidata.org/wiki/Q1320251","display_name":"Intrathecal","level":2,"score":0.4178575873374939},{"id":"https://openalex.org/C90924648","wikidata":"https://www.wikidata.org/wiki/Q120569","display_name":"Gastroenterology","level":1,"score":0.3968634307384491},{"id":"https://openalex.org/C42219234","wikidata":"https://www.wikidata.org/wiki/Q131130","display_name":"Anesthesia","level":1,"score":0.2752453684806824}],"mesh":[],"locations_count":2,"locations":[{"id":"doi:10.5772/intechopen.71970","doi":"https://doi.org/10.5772/intechopen.71970","is_oa":true,"landing_page_url":"https://doi.org/10.5772/intechopen.71970","pdf_url":"https://www.intechopen.com/citation-pdf-url/57792","source":{"id":"https://openalex.org/S4306463447","display_name":"InTech eBooks","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"ebook platform"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Cyclodextrin - A Versatile Ingredient","raw_type":"book-chapter"},{"id":"pmh:oai:biblioboard.com:ad27a768-5ace-4ab0-988a-f4cf905c729e","doi":null,"is_oa":true,"landing_page_url":"https://openresearchlibrary.org/viewer/ad27a768-5ace-4ab0-988a-f4cf905c729e","pdf_url":null,"source":{"id":"https://openalex.org/S4306401131","display_name":"BiblioBoard Library Catalog (Open Research Library)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"MODID-6d55e02e354:IntechOpen","raw_type":"CHAPTER"}],"best_oa_location":{"id":"doi:10.5772/intechopen.71970","doi":"https://doi.org/10.5772/intechopen.71970","is_oa":true,"landing_page_url":"https://doi.org/10.5772/intechopen.71970","pdf_url":"https://www.intechopen.com/citation-pdf-url/57792","source":{"id":"https://openalex.org/S4306463447","display_name":"InTech eBooks","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":null,"host_organization_name":null,"host_organization_lineage":[],"host_organization_lineage_names":[],"type":"ebook platform"},"license":"cc-by","license_id":"https://openalex.org/licenses/cc-by","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Cyclodextrin - A Versatile Ingredient","raw_type":"book-chapter"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":1.0}],"study_designs":[],"awards":[],"funders":[],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W2802807799.pdf","grobid_xml":"https://content.openalex.org/works/W2802807799.grobid-xml"},"referenced_works_count":41,"referenced_works":["https://openalex.org/W566428193","https://openalex.org/W1907295175","https://openalex.org/W1940421635","https://openalex.org/W1965969890","https://openalex.org/W1966095387","https://openalex.org/W1986849641","https://openalex.org/W2003037431","https://openalex.org/W2003672470","https://openalex.org/W2021248130","https://openalex.org/W2028839552","https://openalex.org/W2036164962","https://openalex.org/W2037942173","https://openalex.org/W2040725099","https://openalex.org/W2061330453","https://openalex.org/W2068262899","https://openalex.org/W2078583436","https://openalex.org/W2085578246","https://openalex.org/W2093934810","https://openalex.org/W2094047022","https://openalex.org/W2106113621","https://openalex.org/W2125802191","https://openalex.org/W2135711258","https://openalex.org/W2140479498","https://openalex.org/W2143203936","https://openalex.org/W2147612731","https://openalex.org/W2158140506","https://openalex.org/W2161777076","https://openalex.org/W2162400220","https://openalex.org/W2164903672","https://openalex.org/W2221323276","https://openalex.org/W2237222021","https://openalex.org/W2246776136","https://openalex.org/W2339299959","https://openalex.org/W2343122611","https://openalex.org/W2358799891","https://openalex.org/W2397897146","https://openalex.org/W2523819681","https://openalex.org/W2547028635","https://openalex.org/W2584314070","https://openalex.org/W2606021369","https://openalex.org/W2744065025"],"related_works":["https://openalex.org/W2584314070","https://openalex.org/W2744065025","https://openalex.org/W3177306733","https://openalex.org/W2021248130","https://openalex.org/W2981132400","https://openalex.org/W1907295175","https://openalex.org/W2782551852","https://openalex.org/W3084201033","https://openalex.org/W2085578246","https://openalex.org/W4382602787"],"abstract_inverted_index":{"Niemann-Pick":[0],"disease":[1],"type":[2],"C":[3],"(NPD-C)":[4],"is":[5,154],"a":[6,12,28,62,116,133,144],"rare":[7],"neurodegenerative":[8],"disorder":[9],"characterized":[10],"by":[11],"lysosomal":[13],"storage":[14],"disorder.":[15],"Treatment":[16],"has":[17,44],"been":[18,45,172],"supportive":[19],"and":[20,36,181],"symptomatic.":[21],"In":[22],"animal":[23],"studies,":[24],"2-hydroxypropyl-\u03b2-cyclodextrin":[25],"(HP-\u03b2-CD)":[26],"showed":[27],"significant":[29],"decrease":[30],"in":[31,47,65,104,128,174,194],"cerebellar":[32],"damage,":[33],"neurological":[34,66,98],"progression,":[35],"higher":[37,87],"lifespan.":[38],"Based":[39],"on":[40],"these":[41],"results,":[42],"HP-\u03b2-CD":[43,60,85,111,137,187],"tested":[46],"NPD-C":[48,94,126],"patients":[49,130],"for":[50],"last":[51],"8":[52],"years.":[53],"The":[54,77,113,148,183],"first":[55],"compassionate":[56],"uses":[57],"of":[58,73,84,93,97,110,115,125,136,151,159,197],"intravenous":[59],"obtained":[61],"limited":[63],"improvement":[64,90],"symptoms,":[67],"probably":[68],"associated":[69],"to":[70,106,143],"the":[71,74,108,123,157,162,195],"non-permeation":[72],"blood-brain":[75],"barrier.":[76],"change":[78],"or":[79,91],"combination":[80],"with":[81,132,141,176,186],"intrathecal":[82,138],"administrations":[83],"achieved":[86],"benefits,":[88],"especially":[89],"stabilization":[92],"progression.":[95],"Biomarkers":[96],"cholesterol":[99],"homeostasis":[100],"are":[101],"being":[102,156],"investigated":[103],"order":[105],"quantify":[107],"response":[109],"treatment.":[112],"results":[114],"clinical":[117],"trial":[118],"recently":[119],"published":[120],"have":[121,171],"reproduced":[122],"slowing":[124],"progression":[127],"14":[129],"treated":[131],"dose-escalation":[134],"protocol":[135],"monthly":[139],"infusions,":[140],"respect":[142],"historical":[145],"comparison":[146],"cohort.":[147],"safety":[149],"profile":[150],"this":[152],"therapy":[153],"acceptable,":[155],"loss":[158],"hearing":[160],"as":[161],"most":[163],"frequent":[164],"adverse":[165],"event.":[166],"However,":[167],"some":[168],"severe":[169],"toxicities":[170],"reported":[173],"relation":[175],"HP-\u03b2-CD,":[177],"including":[178],"chemical":[179],"meningitis":[180],"fever.":[182],"short":[184],"experience":[185],"suggested":[188],"that":[189],"it":[190],"could":[191],"be":[192],"effective":[193],"management":[196],"NPD-C.":[198]},"counts_by_year":[{"year":2025,"cited_by_count":1},{"year":2024,"cited_by_count":1},{"year":2021,"cited_by_count":1},{"year":2019,"cited_by_count":1}],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2018-05-17T00:00:00"}
