K
Kenneth J. Valenzano
Researcher at Amicus Therapeutics
Publications - 57
Citations - 2263
Kenneth J. Valenzano is an academic researcher from Amicus Therapeutics. The author has contributed to research in topics: Pharmacological chaperone & Fabry disease. The author has an hindex of 21, co-authored 57 publications receiving 2037 citations.
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Journal ArticleDOI
N-(4-Tertiarybutylphenyl)-4-(3-cholorphyridin-2-yl)tetrahydropyrazine -1(2H)-carbox-amide (BCTC), a novel, orally effective vanilloid receptor 1 antagonist with analgesic properties: II. in vivo characterization in rat models of inflammatory and neuropathic pain.
James D. Pomonis,James E. Harrison,Lilly Mark,David R. Bristol,Kenneth J. Valenzano,Katharine Walker +5 more
TL;DR: The effects of BCTC on acute, inflammatory, and neuropathic pain in rats suggest a role for VR1 in persistent and chronic pain arising from inflammation or nerve injury.
Journal ArticleDOI
N-(4-Tertiarybutylphenyl)-4-(3-chloropyridin-2-yl)tetrahydropyrazine -1(2H)-carbox-amide (BCTC), a Novel, Orally Effective Vanilloid Receptor 1 Antagonist with Analgesic Properties: I. In Vitro Characterization and Pharmacokinetic Properties
Kenneth J. Valenzano,Elfrida R. Grant,Gang Wu,Mohamed Hachicha,Lori Schmid,Laykea Tafesse,Qun Sun,Yakov Rotshteyn,Joseph Francis,James T. Limberis,Shiazah Malik,Edward R. Whittemore,D. Dianne Hodges +12 more
TL;DR: In vitro pharmacology of a high potency, selective VR1 antagonist that, unlike capsazepine, has potent blocking effects on low pH-induced activation of rat VR1 is described, making it a more suitable candidate for testing the role played by VR1 in rat models of human disease.
Journal ArticleDOI
Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders
TL;DR: PCT is an emerging approach based on small-molecule ligands that selectively bind and stabilize mutant enzymes, increase their cellular levels, and improve lysosomal trafficking and activity that shows advantages, particularly in terms of oral administration, broad biodistribution, and positive impact on patients' quality of life.
Journal ArticleDOI
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.
Journal ArticleDOI
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat.
Elfrida R. Benjamin,Maria Cecilia Della Valle,Xiaoyang Wu,Evan Katz,Farhana Pruthi,Sarah Bond,Benjamin Bronfin,Hadis Williams,Julie Yu,Daniel G. Bichet,Dominique P. Germain,Roberto Giugliani,Derralynn Hughes,Raphael Schiffmann,William R. Wilcox,Robert J. Desnick,John Kirk,Jay A. Barth,Carrolee Barlow,Kenneth J. Valenzano,Jeff Castelli,David J. Lockhart +21 more
TL;DR: The GLP HEK assay is a clinically validated method of identifying male and female Fabry patients for treatment with migalastat, a pharmacological chaperone that binds to specific mutant forms of α-galactosidase A to restore lysosomal activity.