Clinical studies in lysosomal storage diseases: Past, present, and future

by Boudes, Pol F
Published 2013

A Pharmacogenetic Approach to Identify Mutant Forms of α-Galactosidase A that Respond to a Pharmacological Chaperone for Fabry Disease

by Wu, Xiaoyang, Katz, Evan, Valle, Maria Cecilia Della, Mascioli, Kirsten, Flanagan, John J, Castelli, Jeffrey P, Schiffmann, Raphael, Boudes, Pol, Lockhart, David J, Valenzano, Kenneth J, Benjamin, Elfrida R
Published 2011

Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb3) in Fabry Transgenic Mice and in the Plasma of Fabry Patients

by Young-Gqamana, Brandy, Brignol, Nastry, Chang, Hui-Hwa, Khanna, Richie, Soska, Rebecca, Fuller, Maria, Sitaraman, Sheela A., Germain, Dominique P., Giugliani, Roberto, Hughes, Derralynn A., Mehta, Atul, Nicholls, Kathy, Boudes, Pol, Lockhart, David J., Valenzano, Kenneth J., Benjamin, Elfrida R.
Published 2013

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinic...

by Germain, Dominique P, Giugliani, Roberto, Hughes, Derralynn A, Mehta, Atul, Nicholls, Kathy, Barisoni, Laura, Jennette, Charles J, Bragat, Alexander, Castelli, Jeff, Sitaraman, Sheela, Lockhart, David J, Boudes, Pol F
Published 2012

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

by Warnock, David G., Bichet, Daniel G., Holida, Myrl, Goker-Alpan, Ozlem, Nicholls, Kathy, Thomas, Mark, Eyskens, Francois, Shankar, Suma, Adera, Mathews, Sitaraman, Sheela, Khanna, Richie, Flanagan, John J., Wustman, Brandon A., Barth, Jay, Barlow, Carrolee, Valenzano, Kenneth J., Lockhart, David J., Boudes, Pol, Johnson, Franklin K.
Published 2015

A Randomized, Double‐Blind, Active‐ and Placebo‐Controlled Efficacy and Safety Study of Arhalofenate for Reducing Flare in Patients With Gout

by Poiley, Jeffrey, Steinberg, Alexandra S., Choi, Yun‐Jung, Davis, Charles S., Martin, Robert L., McWherter, Charles A., Boudes, Pol F., Bays, H., Bolshoun, D., Bolster, E., Cheung, D., Cone, C., DeGarmo, R., Earl, J., El Asmar, I., Felber, D., Fitz‐Patrick, D., Gaffo, A., Gottschlich, G., Greenwald, J., Griffin, C., Guice, M., Harper, W., Hill, J., Huffman, C., Huling, R., Johnson, F., Kafka, S., Keane, J., Kirby, W., Kirstein, J., Kolettis, E., Lefebvre, G., Mabaquiao, A., Maynard, K., McIlwain, H., McNeill, R., Mehta, D., Montgomery, R., Morgan, C., Poiley, J., Powell, S., Radbill, K., Reschak, M., Surowitz, R., Tarro, J., Turner, M., Velazquez, F., Watkins, L., White, J., Williams, H., Wilson, J., Zaidi, F., Henein, S., Toma, A., Burtchuladze, T., Kartvelishvili, E., Kilasonia, L., Lagvilava, L., Shalamberidze, L., Tsiskarishvili, N.
Published 2016