{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,17]],"date-time":"2026-08-17T14:50:13Z","timestamp":1786978213982,"version":"build-2736575974"},"reference-count":20,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2025,11,17]],"date-time":"2025-11-17T00:00:00Z","timestamp":1763337600000},"content-version":"vor","delay-in-days":16,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Molec Gen &amp;amp; Gen Med"],"published-print":{"date-parts":[[2025,11]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Purpose<\/jats:title>\n                    <jats:p>To evaluate the disease biomarker response of venglustat in patients with Fabry disease (FD), utilizing data from a single\u2010arm phase 2 study of venglustat and a placebo\u2010controlled phase 3 study of agalsidase beta through historical control and case\u2010matched analyses.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Eleven venglustat\u2010treated male patients with classic FD in the phase 2 study were matched with placebo\u2010 or agalsidase beta\u2013treated patients from the phase 3 study based on propensity scores at baseline. Changes from baseline in plasma globotriaosylceramide (GL\u20103 or Gb3) concentrations were analyzed at approximately 6\u201336\u2009months.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Venglustat treatment resulted in greater significant reductions in plasma GL\u20103 concentrations at 6\u2009months from baseline vs. placebo (mean difference \u22122.56\u2009\u03bcg\/mL,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.001), and at 24 and 36\u2009months from baseline vs. agalsidase beta (mean difference \u22121.8\u2009\u03bcg\/mL,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.05 and \u22122.35\u2009\u03bcg\/mL,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.01, respectively). GL\u20103 concentrations continued to decline with venglustat for up to 3\u2009years without plateauing.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Venglustat showed significantly greater reductions in plasma GL\u20103 concentrations than placebo after 6\u2009months and agalsidase beta after 24 and 36\u2009months. These findings support the potential of long\u2010term venglustat treatment to reduce GL\u20103 accumulation in patients with classic FD. Further studies are needed to confirm clinical benefit.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mgg3.70152","type":"journal-article","created":{"date-parts":[[2025,11,17]],"date-time":"2025-11-17T11:06:53Z","timestamp":1763377613000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Historical Control Analysis Demonstrates Greater Long\u2010Term Reduction in Plasma Globotriaosylceramide (Gb3) by Venglustat Compared With Placebo or Agalsidase Beta in Male Patients With Classic Fabry Disease"],"prefix":"10.1002","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8355-007X","authenticated-orcid":false,"given":"Dominique P.","family":"Germain","sequence":"first","affiliation":[{"name":"Division of Medical Genetics University of Versailles  Montigny France"},{"name":"French Referral Center for Fabry Disease and Lysosomal Diseases, MetabERN AP\u2010HP Paris\u2010Saclay University  Garches France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pronabesh","family":"DasMahapatra","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shiguang","family":"Liu","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Deegan","sequence":"additional","affiliation":[{"name":"Lysosomal Disorders Unit, Addenbrooke's Hospital Cambridge University Hospitals NHS Foundation Trust  Cambridge UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alberto","family":"Ortiz","sequence":"additional","affiliation":[{"name":"Research Institute\u2010Fundacion Jimenez Diaz Aut\u00f3noma University (IIS\u2010FJD UAM)  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vijay","family":"Modur","sequence":"additional","affiliation":[{"name":"Sanofi  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William R.","family":"Wilcox","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics, Department of Human Genetics Emory University School of Medicine  Atlanta Georgia USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,11,17]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI9711"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.0712309105"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.2119\/molmed.2015.00088"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2016.122"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2023.107585"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2022.11.002"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200107053450102"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-5-30"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.09.007"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgmr.2019.100454"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1510198"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1681\/ASN.2006080816"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2014.120"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-021-01621-y"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.26276"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2018.02.014"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-018-0025-4"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M110.118588"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e3181bb05bb"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1086\/422366"}],"container-title":["Molecular Genetics &amp; Genomic Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mgg3.70152","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,11,26]],"date-time":"2025-11-26T07:06:15Z","timestamp":1764140775000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mgg3.70152"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11]]},"references-count":20,"journal-issue":{"issue":"11","published-print":{"date-parts":[[2025,11]]}},"alternative-id":["10.1002\/mgg3.70152"],"URL":"https:\/\/doi.org\/10.1002\/mgg3.70152","archive":["Portico"],"relation":{},"ISSN":["2324-9269","2324-9269"],"issn-type":[{"value":"2324-9269","type":"print"},{"value":"2324-9269","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,11]]},"assertion":[{"value":"2025-04-14","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-10-10","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-11-17","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70152"}}