Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Authors: Byrne, Barry J., Parenti, Giancarlo, Schoser, Benedikt, van der Ploeg, Ans T., Do, Hung, Fox, Brian, Goldman, Mitchell, Johnson, Franklin K., Kang, Jia, Mehta, Nickita, Mondick, John, Sheikh, M. Osman, Sitaraman Das, Sheela, Tuske, Steven, Brudvig, Jon, Weimer, Jill M., Mozaffar, Tahseen

Source: Frontiers in Neurology; 2025, p1-15, 15p

Subject Terms: GLYCOGEN storage disease type II, ENZYME replacement therapy, LYSOSOMAL storage diseases, PATIENT experience, ENZYME activation, GLYCANS

Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Authors: Byrne, Barry J., Parenti, Giancarlo, Schoser, Benedikt, van der Ploeg, Ans T., Hung Do, Fox, Brian, Goldman, Mitchell, Johnson, Franklin K., Jia Kang, Mehta, Nickita, Mondick, John, Sheikh, M. Osman, Das, Sheela Sitaraman, Tuske, Steven, Brudvig, Jon, Weimer, Jill M., Mozaffar, Tahseen

Source: Frontiers in Neurology; 2024, p1-15, 15p

Subject Terms: GLYCOGEN storage disease type II, ENZYME replacement therapy, LYSOSOMAL storage diseases, PATIENT experience, ENZYME activation, METABOLIC disorders, GLYCANS

Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Authors: Byrne, Barry J., Parenti, Giancarlo, Schoser, Benedikt, van der Ploeg, Ans T., Do, Hung, Fox, Brian, Goldman, Mitchell, Johnson, Franklin K., Kang, Jia, Mehta, Nickita, Mondick, John, Sheikh, M. Osman, Sitaraman Das, Sheela, Tuske, Steven, Brudvig, Jon, Weimer, Jill M., Mozaffar, Tahseen

Source: Frontiers in Neurology; 2025, p1-3, 3p

Subject Terms: GLYCOGEN storage disease type II, LYSOSOMAL storage diseases, ENZYME replacement therapy, TREATMENT effectiveness, SCHOLARLY periodical corrections

Correction of glycogen accumulation in muscle, heart and CNS in a pre-clinical model of hematopoietic stem cell gene therapy for Pompe disease

Authors: Wantuch, Slawomir ^a, Tuske, Steven ^b, Benedetti, Sara ^c, Recchi, Chiara ^a, Heal, Paul ^a, Sagoo, Pervinder ^a, Johnson, Tyler ^b, Mehta, Nickita ^b, Brudvig, Jon ^b, Mavilio, Fulvio ^a, Weimer, Jill ^b, Gaspar, Hubert Bobby ^d

Source: In Molecular Genetics and Metabolism February 2025 144(2)

Structure of IL-17A in Complex with a Potent, Fully Human Neutralizing Antibody

Authors: Gerhardt, Stefan, Abbott, W. Mark, Hargreaves, David, Pauptit, Richard A., Davies, Rick A., Needham, Maurice R.C., Langham, Caroline, Barker, Wendy, Aziz, Azad, Snow, Melanie J., Dawson, Sarah, Welsh, Fraser, Wilkinson, Trevor, Vaugan, Tris, Beste, Gerald, Bishop, Sarah, Popovic, Bojana, Rees, Gareth, Sleeman, Matthew, Tuske, Steven J., Coales, Stephen J., Hamuro, Yoshitomo, Russell, Caroline

Source: In Journal of Molecular Biology 18 December 2009 394(5):905-921

Structural Basis for the Role of the K65R Mutation in HIV-1 Reverse Transcriptase Polymerization, Excision Antagonism, and Tenofovir Resistance

Authors: Das, Kalyan, Bandwar, Rajiv P., White, Kirsten L., Feng, Joy Y., Sarafianos, Stefan G., Tuske, Steven, Tu, Xiongying, Clark, Arthur D., Jr., Boyer, Paul L., Hou, Xiaorong, Gaffney, Barbara L., Jones, Roger A., Miller, Michael D., Hughes, Stephen H., Arnold, Eddy

Source: In Journal of Biological Chemistry 11 December 2009 284(50):35092-35100

The RNA Polymerase “Switch Region” Is a Target for Inhibitors

Authors: Mukhopadhyay, Jayanta, Das, Kalyan, Ismail, Sajida, Koppstein, David, Jang, Minyoung, Hudson, Brian, Sarafianos, Stefan, Tuske, Steven, Patel, Jay, Jansen, Rolf, Irschik, Herbert, Arnold, Eddy, Ebright, Richard H.

Source: In Cell 2008 135(2):295-307

Inhibition of Bacterial RNA Polymerase by Streptolydigin: Stabilization of a Straight-Bridge-Helix Active-Center Conformation

Authors: Tuske, Steven, Sarafianos, Stefan G., Wang, Xinyue, Hudson, Brian, Sineva, Elena, Mukhopadhyay, Jayanta, Birktoft, Jens J., Leroy, Olivier, Ismail, Sajida, Clark, Arthur D., Jr., Dharia, Chhaya, Napoli, Andrew, Laptenko, Oleg, Lee, Jookyung, Borukhov, Sergei, Ebright, Richard H. ^*, Arnold, Eddy ^*

Source: In Cell 26 August 2005 122(4):541-552

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Identification of Pharmacological Chaperones for Gaucher Disease and Characterization of Their Effects on β-Glucocerebrosidase by Hydrogen/Deuterium Exchange Mass Spectrometry.

Authors: Tropak, Michael B., Kornhaber, Gregory J., Rigat, Brigitte A., Maegawa, Gustavo H., Buttner, Justin D., Blanchard, Jan E., Murphy, Cecilia, Tuske, Steven J., Coales, Stephen J., Hamuro, Yoshitomo, Brown, Eric D., Mahuran, Don J.

Source: ChemBioChem; Nov2008, Vol. 9 Issue 16, p2650-2662, 13p

Conformation-Dependent Ligand Regulation of ATP Hydrolysis by Human KSP: Activation of Basal Hydrolysis and Inhibition of Microtubule-Stimulated Hydrolysis by a Single, Small Molecule Modulator.

Authors: Lusong Luo¹, Iusong.luo@gsk.com, Carson, Jeffrey D.¹, Molnar, Kathleen S.², Tuske, Steven J.², Coales, Stephen J.², Hamuro, Yoshitomo², Chiu-Mei Sung³, Sudakin, Valery³, Auger, Kurt R.³, Dhanak, Dashyant⁴, Jackson, Jeffrey A.³, Huang, Pearl S.³, Tummino, Peter J.¹, Copeland, Robert A.¹, robert.a.copeland@gsk.com

Source: Journal of the American Chemical Society; 6/18/2008, Vol. 130 Issue 24, p7584-7591, 7p, 2 Diagrams, 2 Charts, 3 Graphs

Hydrogen/deuterium-exchange (H/D-Ex) of PPARγ LBD in the presence of various modulators.

Authors: Hamuro, Yoshitomo, Coales, Stephen J., Morrow, Jeffrey A., Molnar, Kathleen S., Tuske, Steven J., Southern, Mark R., Griffin, Patrick R.

Source: Protein Science: A Publication of the Protein Society; 2006, Vol. 15 Issue 8, p1883-1892, 10p

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