Kenneth J. Valenzano

Amicus Therapeutics (United States) — United States

Medicine · Health Sciences

36h-index4.6kcitations83works0.02yr avg
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Research Topics

Lysosomal Storage Disorders Research(41), Receptor Mechanisms and Signaling(17), Trypanosoma species research and implications(15), Glycogen Storage Diseases and Myoclonus(15), Neuropeptides and Animal Physiology(13)

Publications83 total

Improved efficacy of a next-generation ERT in murine Pompe disease
JCI Insight·2019· 82 citedOpen Access
A major advance in the search for more effective therapy for Pompe disease
Molecular Genetics and Metabolism·2018
A next-generation Fabry enzyme replacement therapy: A proprietary human α-galactosidase A co-formulated with a pharmacological chaperone, AT1001, shows greater substrate reduction than standard of care in Fabry mice
Molecular Genetics and Metabolism·2018
Glucosylceramide and Glucosylsphingosine Quantitation by Liquid Chromatography-Tandem Mass Spectrometry to Enable In Vivo Preclinical Studies of Neuronopathic Gaucher Disease
Analytical Chemistry·2017· 28 cited
Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α
Molecular Therapy·2017· 44 citedOpen Access
A novel recombinant human acid alpha-glucosidase, ATB200, leads to greater substrate reduction and improvement in Pompe disease-relevant markers compared to alglucosidase alfa in GAA KO mice
Molecular Genetics and Metabolism·2017
Stabilized next-generation recombinant human acid alpha-glucosidase ATB200 clears accumulated glycogen and reverses cellular dysfunction to increase functional muscle strength in a mouse model of Pompe disease
Molecular Genetics and Metabolism·2017
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat
Genetics in Medicine·2016· 207 citedOpen Access
Pharmacological Chaperones as Potential Therapeutics for Lysosomal Storage Disorders: Preclinical Research to Clinical Studies
2016· 3 cited
The validation of pharmacogenetics in the identification of patients with Fabry disease for treatment with migalastat
Molecular Genetics and Metabolism·2016
Comparison of integrated white blood cell alpha-galactosidase a activity exposure between every-other-day orally administered migalastat and biweekly infusions of agalsidase beta or agalsidase alfa
Molecular Genetics and Metabolism·2016· 5 cited
Co-administration of the pharmacological chaperone AT2221 with a proprietary recombinant human acid alfa-glucosidase leads to greater plasma exposure and substrate reduction compared to alglucosidase alfa
Molecular Genetics and Metabolism·2016
Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase
PLoS ONE·2015· 62 citedOpen Access
Coformulation of a Novel Human α -Galactosidase A With the Pharmacological Chaperone AT1001 Leads to Improved Substrate Reduction in Fabry Mice
Molecular Therapy·2015· 36 citedOpen Access
Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders
Molecular Therapy·2015· 224 citedOpen Access
Accurate quantitation of plasma globotriaosylsphingosine (lyso-Gb3) in normal individuals and Fabry disease patients by liquid chromatography–tandem mass spectrometry (LC–MS/MS)
Molecular Genetics and Metabolism·2015
Histological examination of the effect of a highly phosphorylated proprietary recombinant human acid alpha-glucosidase on glycogen reduction in disease-relevant muscles of Pompe mice
Molecular Genetics and Metabolism·2015
Novel recombinant human acid α-glucosidase with optimal glycosylation is significantly better than standard of care enzyme replacement for glycogen clearance in skeletal muscles of GAA knock-out mice
Molecular Genetics and Metabolism·2015· 5 cited
Pharmacological Chaperones to Correct Enzyme Folding, Cellular Trafficking and Lysosomal Activity
2014
The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease
PLoS ONE·2014· 38 citedOpen Access
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Frequent Co-authors

David J. Lockhart(14), Richie Khanna(14), Elfrida R. Benjamin(12), John J. Flanagan(8), Rebecca Soska(8), Jessie Feng(8), Yi Lun(8), Wendy Miller(7), Michelle Frascella(7), Donald J. Kyle(7), Shen Shan(6), Brandon A. Wustman(6), Nastry Brignol(6), Yakov Rotshteyn(5), Robert E. Boyd(5), Adriane Schilling(5), Lee Pellegrino(5), Zhengming Chen(5), Laykea Tafesse(4), Gary Lee(4)