{"id":"https://openalex.org/W3097429941","doi":"https://doi.org/10.1111/cts.12911","title":"Assessment of Target Engagement in a First\u2010in\u2010Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders","display_name":"Assessment of Target Engagement in a First\u2010in\u2010Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders","publication_year":2020,"publication_date":"2020-11-03","ids":{"openalex":"https://openalex.org/W3097429941","doi":"https://doi.org/10.1111/cts.12911","mag":"3097429941","pmid":"https://pubmed.ncbi.nlm.nih.gov/33142037","pmcid":"https://www.ncbi.nlm.nih.gov/pmc/articles/7993281"},"language":"en","primary_location":{"id":"doi:10.1111/cts.12911","doi":"https://doi.org/10.1111/cts.12911","is_oa":true,"landing_page_url":"https://doi.org/10.1111/cts.12911","pdf_url":null,"source":{"id":"https://openalex.org/S106654227","display_name":"Clinical and Translational Science","issn_l":"1752-8054","issn":["1752-8054","1752-8062"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical and Translational Science","raw_type":"journal-article"},"volume":null,"type":"article","is_traditional":true,"indexed_in":["crossref","doaj","pubmed"],"open_access":{"is_oa":true,"oa_status":"gold","oa_url":"https://doi.org/10.1111/cts.12911","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5047505892","display_name":"Martine G\u00e9hin","orcid":"https://orcid.org/0000-0003-0780-1073","observed_orcids":["https://orcid.org/0000-0003-0780-1073"]},"institutions":[{"id":"https://openalex.org/I4405257665","display_name":"Idorsia Pharmaceuticals Ltd (Switzerland)","ror":"https://ror.org/05j00sy30","country_code":"CH","type":"company","lineage":["https://openalex.org/I4210136915","https://openalex.org/I4405257665"]}],"countries":["CH"],"is_corresponding":true,"raw_author_name":"Martine Gehin","raw_affiliation_strings":["Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland","institution_ids":["https://openalex.org/I4405257665"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5081911571","display_name":"Meggane Melchior","orcid":"https://orcid.org/0000-0002-0999-7279","observed_orcids":["https://orcid.org/0000-0002-0999-7279"]},"institutions":[{"id":"https://openalex.org/I4405257665","display_name":"Idorsia Pharmaceuticals Ltd (Switzerland)","ror":"https://ror.org/05j00sy30","country_code":"CH","type":"company","lineage":["https://openalex.org/I4210136915","https://openalex.org/I4405257665"]}],"countries":["CH"],"is_corresponding":false,"raw_author_name":"Meggane Melchior","raw_affiliation_strings":["Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland","institution_ids":["https://openalex.org/I4405257665"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5037490511","display_name":"Richard W.D. Welford","orcid":"https://orcid.org/0000-0003-4498-9888","observed_orcids":["https://orcid.org/0000-0003-4498-9888"]},"institutions":[{"id":"https://openalex.org/I4405257665","display_name":"Idorsia Pharmaceuticals Ltd (Switzerland)","ror":"https://ror.org/05j00sy30","country_code":"CH","type":"company","lineage":["https://openalex.org/I4210136915","https://openalex.org/I4405257665"]}],"countries":["CH"],"is_corresponding":false,"raw_author_name":"Richard W.D. Welford","raw_affiliation_strings":["Drug Discovery, Translational Biomarkers, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Drug Discovery, Translational Biomarkers, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland","institution_ids":["https://openalex.org/I4405257665"]}]},{"author_position":"middle","author":{"id":"https://openalex.org/A5085651386","display_name":"Patricia N. Sidharta","orcid":"https://orcid.org/0000-0001-7581-4525","observed_orcids":["https://orcid.org/0000-0001-7581-4525"]},"institutions":[{"id":"https://openalex.org/I4405257665","display_name":"Idorsia Pharmaceuticals Ltd (Switzerland)","ror":"https://ror.org/05j00sy30","country_code":"CH","type":"company","lineage":["https://openalex.org/I4210136915","https://openalex.org/I4405257665"]}],"countries":["CH"],"is_corresponding":false,"raw_author_name":"Patricia N. Sidharta","raw_affiliation_strings":["Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland","institution_ids":["https://openalex.org/I4405257665"]}]},{"author_position":"last","author":{"id":"https://openalex.org/A5058093690","display_name":"Jasper Dingemanse","orcid":"https://orcid.org/0000-0002-4083-5817","observed_orcids":["https://orcid.org/0000-0002-4083-5817"]},"institutions":[{"id":"https://openalex.org/I4405257665","display_name":"Idorsia Pharmaceuticals Ltd (Switzerland)","ror":"https://ror.org/05j00sy30","country_code":"CH","type":"company","lineage":["https://openalex.org/I4210136915","https://openalex.org/I4405257665"]}],"countries":["CH"],"is_corresponding":false,"raw_author_name":"Jasper Dingemanse","raw_affiliation_strings":["Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland"],"raw_orcid":null,"affiliations":[{"raw_affiliation_string":"Clinical Pharmacology, Idorsia Pharmaceuticals Ltd, Allschwil, Switzerland","institution_ids":["https://openalex.org/I4405257665"]}]}],"institutions":[],"countries_distinct_count":1,"institutions_distinct_count":1,"corresponding_author_ids":["https://openalex.org/A5047505892"],"corresponding_institution_ids":["https://openalex.org/I4405257665"],"apc_list":{"value":3000,"currency":"USD","value_usd":3000},"apc_paid":{"value":3000,"currency":"USD","value_usd":3000},"fwci":0.8038,"has_fulltext":true,"cited_by_count":11,"citation_normalized_percentile":{"value":0.69146797,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":{"min":88,"max":98},"biblio":{"volume":"14","issue":"2","first_page":"558","last_page":"567"},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10945","display_name":"Lysosomal Storage Disorders Research","score":0.9994999766349792,"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":0.9994999766349792,"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":0.00019999999494757503,"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/T11308","display_name":"Sphingolipid Metabolism and Signaling","score":0.0,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"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"}}],"keywords":[{"id":"https://openalex.org/keywords/iminosugars","display_name":"iminosugars","score":0.8190000057220459},{"id":"https://openalex.org/keywords/lysosomal-storage-disorders","display_name":"lysosomal storage disease","score":0.9010000228881836},{"id":"https://openalex.org/keywords/glucosylceramide-synthase-inhibition","display_name":"glucosylceramide synthase inhibition","score":0.9580000042915344},{"id":"https://openalex.org/keywords/glucosylceramide","display_name":"glucosylceramide","score":0.9399999976158142},{"id":"https://openalex.org/keywords/lactosylceramide","display_name":"lactosylceramide","score":0.9039999842643738},{"id":"https://openalex.org/keywords/globotriaosylceramide","display_name":"globotriaosylceramide","score":0.9990000128746033},{"id":"https://openalex.org/keywords/glycosphingolipids","display_name":"glycosphingolipids","score":0.8399999737739563},{"id":"https://openalex.org/keywords/pharmacokinetics","display_name":"pharmacokinetics","score":0.8629999756813049},{"id":"https://openalex.org/keywords/pharmacodynamics","display_name":"pharmacodynamics","score":0.9990000128746033},{"id":"https://openalex.org/keywords/target-engagement","display_name":"target engagement","score":0.7350000143051147},{"id":"https://openalex.org/keywords/tolerability","display_name":"tolerability","score":0.8970000147819519},{"id":"https://openalex.org/keywords/dose-escalation","display_name":"dose escalation","score":0.39800000190734863}],"concepts":[{"id":"https://openalex.org/C2910328161","wikidata":"https://www.wikidata.org/wiki/Q3215908","display_name":"Lactosylceramide","level":3,"score":0.856464684009552},{"id":"https://openalex.org/C2778375690","wikidata":"https://www.wikidata.org/wiki/Q7814214","display_name":"Tolerability","level":3,"score":0.8256449103355408},{"id":"https://openalex.org/C112705442","wikidata":"https://www.wikidata.org/wiki/Q323936","display_name":"Pharmacokinetics","level":2,"score":0.6814258098602295},{"id":"https://openalex.org/C111113717","wikidata":"https://www.wikidata.org/wiki/Q725307","display_name":"Pharmacodynamics","level":3,"score":0.6284142732620239},{"id":"https://openalex.org/C2777687718","wikidata":"https://www.wikidata.org/wiki/Q410539","display_name":"Globotriaosylceramide","level":4,"score":0.6187869310379028},{"id":"https://openalex.org/C98274493","wikidata":"https://www.wikidata.org/wiki/Q128406","display_name":"Pharmacology","level":1,"score":0.607556939125061},{"id":"https://openalex.org/C71924100","wikidata":"https://www.wikidata.org/wiki/Q11190","display_name":"Medicine","level":0,"score":0.5778629779815674},{"id":"https://openalex.org/C2777288759","wikidata":"https://www.wikidata.org/wiki/Q4164182","display_name":"Dosing","level":2,"score":0.5623825788497925},{"id":"https://openalex.org/C27081682","wikidata":"https://www.wikidata.org/wiki/Q269829","display_name":"Placebo","level":3,"score":0.5388540625572205},{"id":"https://openalex.org/C2776931407","wikidata":"https://www.wikidata.org/wiki/Q418892","display_name":"Glycosphingolipid","level":2,"score":0.4860840141773224},{"id":"https://openalex.org/C2776792218","wikidata":"https://www.wikidata.org/wiki/Q18351124","display_name":"Iminosugar","level":3,"score":0.45503541827201843},{"id":"https://openalex.org/C126322002","wikidata":"https://www.wikidata.org/wiki/Q11180","display_name":"Internal medicine","level":1,"score":0.4257645010948181},{"id":"https://openalex.org/C2777404818","wikidata":"https://www.wikidata.org/wiki/Q615645","display_name":"Fabry disease","level":3,"score":0.38461822271347046},{"id":"https://openalex.org/C134018914","wikidata":"https://www.wikidata.org/wiki/Q162606","display_name":"Endocrinology","level":1,"score":0.35027098655700684},{"id":"https://openalex.org/C185592680","wikidata":"https://www.wikidata.org/wiki/Q2329","display_name":"Chemistry","level":0,"score":0.25032514333724976},{"id":"https://openalex.org/C197934379","wikidata":"https://www.wikidata.org/wiki/Q2047938","display_name":"Adverse effect","level":2,"score":0.2218654453754425},{"id":"https://openalex.org/C55493867","wikidata":"https://www.wikidata.org/wiki/Q7094","display_name":"Biochemistry","level":1,"score":0.12419220805168152},{"id":"https://openalex.org/C2779134260","wikidata":"https://www.wikidata.org/wiki/Q12136","display_name":"Disease","level":2,"score":0.12069594860076904},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.09938943386077881},{"id":"https://openalex.org/C142724271","wikidata":"https://www.wikidata.org/wiki/Q7208","display_name":"Pathology","level":1,"score":0.08852973580360413},{"id":"https://openalex.org/C181199279","wikidata":"https://www.wikidata.org/wiki/Q8047","display_name":"Enzyme","level":2,"score":0.07252305746078491},{"id":"https://openalex.org/C22222904","wikidata":"https://www.wikidata.org/wiki/Q422882","display_name":"Glycolipid","level":2,"score":0.0},{"id":"https://openalex.org/C204787440","wikidata":"https://www.wikidata.org/wiki/Q188504","display_name":"Alternative medicine","level":2,"score":0.0}],"mesh":[{"descriptor_ui":"D000284","descriptor_name":"Administration, Oral","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D000328","descriptor_name":"Adult","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004305","descriptor_name":"Dose-Response Relationship, Drug","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D004311","descriptor_name":"Double-Blind Method","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005260","descriptor_name":"Female","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D005962","descriptor_name":"Glucosylceramidase","qualifier_ui":"Q000037","qualifier_name":"antagonists & inhibitors","is_major_topic":true},{"descriptor_ui":"D005964","descriptor_name":"Glucosyltransferases","qualifier_ui":"Q000037","qualifier_name":"antagonists & inhibitors","is_major_topic":true},{"descriptor_ui":"D006207","descriptor_name":"Half-Life","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D006801","descriptor_name":"Humans","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008297","descriptor_name":"Male","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D008875","descriptor_name":"Middle Aged","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D010880","descriptor_name":"Piperidines","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D010880","descriptor_name":"Piperidines","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D010880","descriptor_name":"Piperidines","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D010919","descriptor_name":"Placebos","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":false},{"descriptor_ui":"D010919","descriptor_name":"Placebos","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D016464","descriptor_name":"Lysosomal Storage Diseases","qualifier_ui":"Q000188","qualifier_name":"drug therapy","is_major_topic":true},{"descriptor_ui":"D050111","descriptor_name":"Imino Sugars","qualifier_ui":"Q000008","qualifier_name":"administration & dosage","is_major_topic":true},{"descriptor_ui":"D050111","descriptor_name":"Imino Sugars","qualifier_ui":"Q000009","qualifier_name":"adverse effects","is_major_topic":false},{"descriptor_ui":"D050111","descriptor_name":"Imino Sugars","qualifier_ui":"Q000493","qualifier_name":"pharmacokinetics","is_major_topic":false},{"descriptor_ui":"D055815","descriptor_name":"Young Adult","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false},{"descriptor_ui":"D064368","descriptor_name":"Healthy Volunteers","qualifier_ui":null,"qualifier_name":null,"is_major_topic":false}],"locations_count":4,"locations":[{"id":"doi:10.1111/cts.12911","doi":"https://doi.org/10.1111/cts.12911","is_oa":true,"landing_page_url":"https://doi.org/10.1111/cts.12911","pdf_url":null,"source":{"id":"https://openalex.org/S106654227","display_name":"Clinical and Translational Science","issn_l":"1752-8054","issn":["1752-8054","1752-8062"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical and Translational Science","raw_type":"journal-article"},{"id":"pmh:oai:pubmedcentral.nih.gov:7993281","doi":null,"is_oa":true,"landing_page_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC7993281/","pdf_url":"https://pmc.ncbi.nlm.nih.gov/articles/PMC7993281/pdf/","source":{"id":"https://openalex.org/S2764455111","display_name":"PubMed Central","issn_l":null,"issn":null,"is_oa":true,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clin Transl Sci","raw_type":"Text"},{"id":"pmid:33142037","doi":null,"is_oa":false,"landing_page_url":"https://pubmed.ncbi.nlm.nih.gov/33142037","pdf_url":null,"source":{"id":"https://openalex.org/S4306525036","display_name":"PubMed","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I1299303238","host_organization_name":"National Institutes of Health","host_organization_lineage":["https://openalex.org/I1299303238"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical and translational science","raw_type":"Clinical Trial, Phase I"},{"id":"pmh:oai:doaj.org/article:ed04fd0658df4e82b8e0c214c3adc261","doi":null,"is_oa":true,"landing_page_url":"https://doaj.org/article/ed04fd0658df4e82b8e0c214c3adc261","pdf_url":null,"source":{"id":"https://openalex.org/S4306401280","display_name":"DOAJ (DOAJ: Directory of Open Access Journals)","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-sa","license_id":"https://openalex.org/licenses/cc-by-sa","version":"submittedVersion","is_accepted":false,"is_published":false,"raw_source_name":"Clinical and Translational Science, Vol 14, Iss 2, Pp 558-567 (2021)","raw_type":"article"}],"best_oa_location":{"id":"doi:10.1111/cts.12911","doi":"https://doi.org/10.1111/cts.12911","is_oa":true,"landing_page_url":"https://doi.org/10.1111/cts.12911","pdf_url":null,"source":{"id":"https://openalex.org/S106654227","display_name":"Clinical and Translational Science","issn_l":"1752-8054","issn":["1752-8054","1752-8062"],"is_oa":true,"is_in_doaj":true,"is_core":true,"listed_in":["cwts-core","doaj","dongbi-c","doyens","jufo-2","ki-jl-1","medline","norway-1","oarl","poland-70","russia-white-list-1","vabb-shw"],"host_organization":"https://openalex.org/P4310320595","host_organization_name":"Wiley","host_organization_lineage":["https://openalex.org/P4310320595"],"host_organization_lineage_names":["Wiley"],"type":"journal"},"license":"cc-by-nc-nd","license_id":"https://openalex.org/licenses/cc-by-nc-nd","version":"publishedVersion","is_accepted":true,"is_published":true,"raw_source_name":"Clinical and Translational Science","raw_type":"journal-article"},"sustainable_development_goals":[{"display_name":"Good health and well-being","id":"https://metadata.un.org/sdg/3","score":0.9940000176429749}],"study_designs":[{"display_name":"Clinical Trial","id":"https://openalex.org/study-designs/clinical-trial"}],"awards":[],"funders":[{"id":"https://openalex.org/F4320329429","display_name":"Idorsia Pharmaceuticals","ror":"https://ror.org/03va9ng43"}],"has_content":{"grobid_xml":true,"pdf":true},"content_urls":{"pdf":"https://content.openalex.org/works/W3097429941.pdf","grobid_xml":"https://content.openalex.org/works/W3097429941.grobid-xml"},"referenced_works_count":37,"referenced_works":["https://openalex.org/W1578426620","https://openalex.org/W1775418466","https://openalex.org/W1825944839","https://openalex.org/W1970333224","https://openalex.org/W1972455024","https://openalex.org/W1973771269","https://openalex.org/W1981747359","https://openalex.org/W2022636376","https://openalex.org/W2026842175","https://openalex.org/W2031328106","https://openalex.org/W2037395785","https://openalex.org/W2039001847","https://openalex.org/W2041175508","https://openalex.org/W2044534774","https://openalex.org/W2068262899","https://openalex.org/W2073433738","https://openalex.org/W2081027701","https://openalex.org/W2111796864","https://openalex.org/W2125281037","https://openalex.org/W2127501479","https://openalex.org/W2135304921","https://openalex.org/W2135424735","https://openalex.org/W2147798737","https://openalex.org/W2170106154","https://openalex.org/W2183225184","https://openalex.org/W2234476209","https://openalex.org/W2341564448","https://openalex.org/W2395282085","https://openalex.org/W2468936663","https://openalex.org/W2480043852","https://openalex.org/W2608651147","https://openalex.org/W2735719527","https://openalex.org/W2739756331","https://openalex.org/W2921423796","https://openalex.org/W2922159284","https://openalex.org/W2948188928","https://openalex.org/W3000003655"],"related_works":["https://openalex.org/W7117298671","https://openalex.org/W2572336419","https://openalex.org/W4389764133","https://openalex.org/W4409499847","https://openalex.org/W3080525978","https://openalex.org/W2132966749","https://openalex.org/W4379260174","https://openalex.org/W4396573997","https://openalex.org/W2044859092","https://openalex.org/W2090051116"],"abstract_inverted_index":{"In":[0,127,139],"this":[1],"first-in-human":[2],"study,":[3,142],"the":[4,101,108,112,116,140,174,176,203,219,236],"tolerability,":[5],"pharmacokinetics":[6],"(PK),":[7],"and":[8,13,26,40,48,59,74,81,94,103,161,193,240],"pharmacodynamics":[9],"(PD)":[10],"of":[11,17,22,111,115,124,157,206],"single":[12],"multiple":[14,60],"oral":[15],"doses":[16,51,61],"sinbaglustat,":[18],"a":[19,121,165],"dual":[20],"inhibitor":[21],"glucosylceramide":[23,158],"synthase":[24],"(GCS)":[25],"non-lysosomal":[27],"glucosyl":[28],"ceramidase":[29],"(GBA2),":[30],"were":[31,45,77,84,145],"investigated":[32],"in":[33,57,72,100,164,235],"healthy":[34],"subjects.":[35],"The":[36,171,223],"single-ascending":[37],"dose":[38,42,110],"(SAD)":[39],"multiple-ascending":[41],"(MAD)":[43],"studies":[44],"randomized,":[46],"double-blind,":[47],"placebo-controlled.":[49],"Single":[50],"from":[52,62],"10":[53],"to":[54,64,87,181,191,202,211,218],"2,000":[55],"mg":[56,66],"men":[58],"30":[63],"1,000":[65],"twice":[67],"daily":[68],"for":[69,238],"7":[70],"days":[71,89],"male":[73],"female":[75,118],"subjects":[76,119],"investigated.":[78],"Tolerability,":[79],"PK,":[80],"PD":[82,183],"data":[83],"collected":[85],"up":[86,217],"3":[88],"after":[90],"(last)":[91],"treatment":[92,186],"administration":[93],"analyzed":[95],"descriptively.":[96],"Sinbaglustat":[97,231],"was":[98,131,179,215],"well-tolerated":[99],"SAD":[102],"MAD":[104,141],"studies,":[105],"however,":[106],"at":[107,197],"highest":[109,220],"MAD,":[113],"three":[114],"four":[117],"presented":[120],"similar":[122],"pattern":[123],"general":[125],"symptoms.":[126],"all":[128],"cohorts,":[129],"sinbaglustat":[130,153,207,214],"rapidly":[132],"absorbed.":[133],"Thereafter,":[134],"plasma":[135,155],"concentrations":[136,156],"decreased":[137,163],"biphasically.":[138],"steady-state":[143],"conditions":[144],"reached":[146],"on":[147,208],"Day":[148],"2":[149],"without":[150],"accumulation.":[151],"During":[152],"treatment,":[154],"(GlcCer),":[159],"lactosylceramide,":[160],"globotriaosylceramide":[162],"dose-dependent":[166],"manner,":[167],"reflecting":[168],"GCS":[169,239],"inhibition.":[170],"more":[172,177],"complex":[173],"glycosphingolipid,":[175],"time":[178],"required":[180],"elicit":[182],"changes.":[184],"After":[185],"stop,":[187],"GlcCer":[188],"levels":[189],"returned":[190],"baseline":[192,196],"increased":[194],"above":[195],"lowest":[198],"doses,":[199],"probably":[200],"due":[201],"higher":[204],"potency":[205],"GBA2":[209],"compared":[210],"GCS.":[212],"Overall,":[213],"welltolerated":[216],"tested":[221],"doses.":[222],"PK":[224],"profile":[225],"is":[226],"compatible":[227],"with":[228],"b.i.d.":[229],"dosing.":[230],"demonstrated":[232],"target":[233],"engagement":[234],"periphery":[237],"GBA2.":[241]},"counts_by_year":[{"year":2026,"cited_by_count":2},{"year":2025,"cited_by_count":5},{"year":2023,"cited_by_count":1},{"year":2022,"cited_by_count":2},{"year":2020,"cited_by_count":1}],"updated_date":"2026-11-11T12:16:26.678958","created_date":"2020-11-09T00:00:00"}