C.W. Pine
Amicus Therapeutics
6 Papers
15 Citations
C.W. Pine is an academic researcher from Amicus Therapeutics. The author has contributed to research in topics: Pharmacological chaperone & Multiple myeloma. The author has an hindex of 4, co-authored 6 publications.
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Papers
Structure of acid beta-glucosidase with pharmacological chaperone provides insight into Gaucher disease.
Raquel L. Lieberman,Brandon Wustman,Pedro Huertas,Pedro Huertas,Allan C. Powe,C.W. Pine,Richie Khanna,Michael G. Schlossmacher,Dagmar Ringe,Dagmar Ringe,Gregory A. Petsko,Gregory A. Petsko +11 more
TL;DR: It is demonstrated that isofagomine (IFG, 1) binds to the GCase active site, and both increases GCase activity in cell lysates and restores lysosomal trafficking in cells containing N370S mutant GCase.
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The pharmacological chaperone 1-deoxygalactonojirimycin increases α-galactosidase A levels in Fabry patient cell lines
Elfrida R. Benjamin,John J. Flanagan,Adriane Schilling,Hui-Hwa Chang,L. Agarwal,Evan Katz,Xiaoyang Wu,C.W. Pine,Brandon W. Wustman,Robert J. Desnick,David J. Lockhart,Kenneth J. Valenzano +11 more
TL;DR: The data indicate that DGJ merits further evaluation as a treatment for patients with Fabry disease with various missense mutations, and elevated GL-3 levels in responsive Fabry fibroblasts were reduced after DGJ incubation, indicating that increased mutant α-Gal A levels can reduce accumulated substrate.
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Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-β-glucosidase
TL;DR: It is reported that IFG has little or no inhibitory activity towards intestinal disaccharidase enzymes, ER alpha-glucosidase II or glucosylceramide synthase at concentrations previously shown to enhance N370S GlcCerase folding and trafficking in Gaucher fibroblasts.
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114. Pharmacological chaperone therapy for Gaucher disease: Mechanism of action, a survey of responsive mutations and phase I clinical trial results
Brandon Wustman,C.W. Pine,Brian Ranes,John J. Flanagan,David Palling,Do Hung,Frank Insinga,Gregory A. Grabowski,Neal J. Weinreb,Gregory M. Pastores,Paul M. Fernhoff,Paige Kaplan,David J. Lockhart +12 more
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Pharmacological Chaperone Therapy for the Treatment of Gaucher Disease: Results of Phase 1 Clinical Trials and a Clinical Ex Vivo Response Study with a Survey of Blood Markers for 53 Gaucher Patients.
Neal J. Weinreb,G.A. Grabowski,G. M. Pastores,Paul M. Fernhoff,Paige Kaplan,David Palling,C.W. Pine,B.E. Raines,F. Insinga,David J. Lockhart,Hung V. Do,Brandon Wustman +11 more
TL;DR: Results show that an imbalance between osteoclast and osteoblast activities may remain even though treatment with imiglucerase, and a dose-dependent increase in GCase levels in white blood cells was observed during the 7 day treatment period.
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