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Drug Evaluations

A systematic review on effectiveness and safety of eliglustat for type 1 Gaucher disease

, MD & , MD PhD

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Umamaheshwar Puppala, Koduri S V Srinivas, K Venkateshwara Reddy, Muralidharan Kaliyaperumal, Raju Doddipalla & Bhaskar Rao Jogi. (2020) Isolation and Characterization of Novel Degradation Products of Eliglustat Tartrate Using 2D-NMR and HRMS: Development and Validation of Stability Indicating RP-UPLC Method for Quantification of Assay and Characterized Impurities. Analytical Chemistry Letters 10:1, pages 1-20.
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Yuta Tanaka, Masaki Seto, Keiko Kakegawa, Kazuaki Takami, Fumiaki Kikuchi, Takeshi Yamamoto, Minoru Nakamura, Masaki Daini, Masataka Murakami, Tomohiro Ohashi, Takahito Kasahara, Junsi Wang, Zenichi Ikeda, Yasufumi Wada, Florian Puenner, Takahiro Fujii, Masakazu Inazuka, Sho Sato, Tomohiko Suzaki, Jeong-Ho Oak, Yuichi Takai, Hiroshi Kohara, Kouya Kimoto, Hideyuki Oki, Satoshi Mikami, Minoru Sasaki & Yuta Tanaka. (2022) Discovery of Brain-Penetrant Glucosylceramide Synthase Inhibitors with a Novel Pharmacophore. Journal of Medicinal Chemistry 65:5, pages 4270-4290.
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Takahiro Fujii, Yuta Tanaka, Hideyuki Oki, Sho Sato, Sachio Shibata, Takamitsu Maru, Yuta Tanaka, Maiko Tanaka & Tomohiro Onishi. (2021) A new brain‐penetrant glucosylceramide synthase inhibitor as potential Therapeutics for Gaucher disease. Journal of Neurochemistry 159:3, pages 543-553.
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Immo Serbian, Erik Prell, Claudia Fischer, Hans-Peter Deigner & René Csuk. (2021) n-Propyl 6-amino-2,6-dideoxy-2,2-difluoro-β-d-glucopyranoside is a good inhibitor for the β-galactosidase from E. coli. Medicinal Chemistry Research 30:5, pages 1099-1107.
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John Marshall, Jennifer B. Nietupski, Hyejung Park, James Cao, Dinesh S. Bangari, Cristina Silvescu, Terry Wilper, Kristen Randall, Drew Tietz, Bing Wang, Xiaoyou Ying, John P. Leonard & Seng H. Cheng. (2019) Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity. Molecular Therapy 27:8, pages 1495-1506.
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Majid Davari, Azita Nabizadeh, Maliheh Kadivar, Akbar Abdollahi Asl & Peymaneh Sarkheil. (2019) Healthcare resource utilization and cost of care for Gaucher patients in Iran. Journal of Diabetes & Metabolic Disorders 18:1, pages 127-132.
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Umamaheshwar Puppala, Koduri S.V. Srinivas, K. Venkateshwara Reddy, Muralidharan Kaliyaperumal & Srinivasa Rao Pakalapati. (2019) Enantiospecific UPC2 SFC-MS method for separation and quantification of R & S-Eliglustat tartrate in presence of its stereo isomers and degradation impurities. Materials Today: Proceedings 19, pages 420-428.
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Bouwien E. Smid, Maria J. Ferraz, Marri Verhoek, Mina Mirzaian, Patrick Wisse, Herman S. Overkleeft, Carla E. Hollak & Johannes M. Aerts. (2016) Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients. Orphanet Journal of Rare Diseases 11:1.
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John Marshall, Ying Sun, Dinesh S Bangari, Eva Budman, Hyejung Park, Jennifer B Nietupski, Amy Allaire, Mary A Cromwell, Bing Wang, Gregory A Grabowski, John P Leonard & Seng H Cheng. (2016) CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease. Molecular Therapy 24:6, pages 1019-1029.
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Karen M. Ashe, Eva Budman, Dinesh S. Bangari, Craig S. Siegel, Jennifer B. Nietupski, Bing Wang, Robert J. Desnick, Ronald K. Scheule, John P. Leonard, Seng H. Cheng & John Marshall. (2015) Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease. Molecular Medicine 21:1, pages 389-399.
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