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Sinbaglustat ameliorates disease pathology in a murine model of GM1 gangliosidosis without affecting CNS ganglioside levels.

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Title: Sinbaglustat ameliorates disease pathology in a murine model of GM1 gangliosidosis without affecting CNS ganglioside levels.
Authors: Wannemacher R; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany., Jubran-Rudolf L; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany., Zdora I; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany., Leitzen E; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany., Rohn K; Department of Biometry, Epidemiology and Data processing, University of Veterinary Medicine Hannover, Bünteweg 12, 30559 Hannover, Germany., Sippel V; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland., Paschen C; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland., Blattmann P; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland., Baumgärtner W; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany. Electronic address: wolfgang.baumgaertner@tiho-hannover.de., Gerhauser I; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany., Steiner MA; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland.
Source: Neurobiology of disease [Neurobiol Dis] 2025 Jun 15; Vol. 210, pp. 106917. Date of Electronic Publication: 2025 Apr 16.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Academic Press Country of Publication: United States NLM ID: 9500169 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1095-953X (Electronic) Linking ISSN: 09699961 NLM ISO Abbreviation: Neurobiol Dis Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Sinbaglustat ameliorates disease pathology in a murine model of G<subscript>M1</subscript> gangliosidosis without affecting CNS ganglioside levels.
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  Data: <searchLink fieldCode="AU" term="%22Wannemacher+R%22">Wannemacher R</searchLink>; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Jubran-Rudolf+L%22">Jubran-Rudolf L</searchLink>; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Zdora+I%22">Zdora I</searchLink>; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Leitzen+E%22">Leitzen E</searchLink>; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Rohn+K%22">Rohn K</searchLink>; Department of Biometry, Epidemiology and Data processing, University of Veterinary Medicine Hannover, Bünteweg 12, 30559 Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Sippel+V%22">Sippel V</searchLink>; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland.<br /><searchLink fieldCode="AU" term="%22Paschen+C%22">Paschen C</searchLink>; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland.<br /><searchLink fieldCode="AU" term="%22Blattmann+P%22">Blattmann P</searchLink>; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland.<br /><searchLink fieldCode="AU" term="%22Baumgärtner+W%22">Baumgärtner W</searchLink>; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany. Electronic address: wolfgang.baumgaertner@tiho-hannover.de.<br /><searchLink fieldCode="AU" term="%22Gerhauser+I%22">Gerhauser I</searchLink>; Department of Pathology, University of Veterinary Medicine Hannover, Foundation, Bünteweg 17, 30559 Hannover, Germany; Center for Systems Neuroscience, Hannover, Germany.<br /><searchLink fieldCode="AU" term="%22Steiner+MA%22">Steiner MA</searchLink>; Idorsia Pharmaceuticals Ltd., Hegenheimermattweg 91, 4123 Allschwil, Switzerland.
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  Data: <searchLink fieldCode="JN" term="%229500169%22">Neurobiology of disease</searchLink> [Neurobiol Dis] 2025 Jun 15; Vol. 210, pp. 106917. <i>Date of Electronic Publication: </i>2025 Apr 16.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Academic+Press%22">Academic Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>9500169 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1095-953X (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2209699961%22">09699961 </searchLink><i>NLM ISO Abbreviation: </i>Neurobiol Dis <i>Subsets: </i>MEDLINE
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