Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease von Ivanova, Margarita M, Dao, Julia, Kasaci, Neil, Adewale, Benjamin, Nazari, Shaista, Noll, Lauren, Fikry, Jacqueline, Sanati, Armaghan Hafez, Goker-Alpan, Ozlem

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Wnt signaling pathway inhibitors, sclerostin and DKK-1, correlate with pain and bone pathology in patients with Gaucher disease von Ivanova, Margarita M, Dao, Julia, Kasaci, Neil, Friedman, Andrew, Noll, Lauren, Goker-Alpan, Ozlem

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Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease von Margarita M Ivanova, Julia Dao, Neil Kasaci, Benjamin Adewale, Shaista Nazari, Lauren Noll, Jacqueline Fikry, Armaghan Hafez Sanati, Ozlem Goker-Alpan

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Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial von Diaz-Manera, Jordi, Kishnani, Priya S, Ladha, Shafeeq, Mozaffar, Tahseen, Straub, Volker, Berger, Kenneth I, Chien, Yin-Hsiu, Day, John W, Illarioshkin, Sergey, Roberts, Mark, Attarian, Shahram, Borges, Joao Lindolfo, Choi, Young Chul, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, Haack, Kristina An, Hug, Christopher, Huynh-Ba, Olivier, Johnson, Judith, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M, Behin, Anthony, Boentert, Matthias, Carvalho, Gerson, Chahin, Nizar, Deegan, Patrick, Duval, Fanny, Genge, Angela, Gutmann, Ludwig, Henderson, Robert D, Hennermann, Julia B, Hiwot, Tarekegn, Hughes, Derralynn, Karaa, Amel, Komaki, Hirofumi, Longo, Nicola, Malinova, Vera, Maxit, Clarisa, Mengel, Eugen, Moggio, Maurizio Gualtiero, Mongini, Tiziana Enrica, Nascimento Osorio, Andres, Noury, Jean-Baptiste, Oliveira, Acary Souza Bulle, Pena, Loren, Remiche, Gauthier, Sciacco, Monica, Smith, Cheryl, Stulnig, Thomas, Taithe, Frederic, Tarnopolsky, Mark, Young, Peter, Alonso-Pérez, Jorge, Altemus, Patricia, Aubé-Nathier, Anne-Catherine, Avelar, Jennifer B, Billy, Jenny, Boschi, Silvia, Carrera Garcia, Laura, Chase, Lauren, Danjoux, Loïc, Davion, Jean-Baptiste, DeArmey, Stephanie, Gandolfo, Eve, Grosz, Zoltan, Guellec, Dewi, Guettsches, Anne-Katrin, Guglieri, Michela, Hatcher, Erin, Hufgard-Leitner, Miriam, Klyushnikov, Sergey A., Langton, Jacqui, Linková, Lenka, Mavroudakis, Nicolas, Mazurová, Stella, Mori, Madoka, Müller-Miny, Louisa, Natera-de Benito, Daniel, Neel, Robert, Niizawa, Gabriela A, Noll, Lauren, Pasnoor, Mamatha, Questienne, Claire, Ramos Lopes, Margarida, Riedl, Michaela, Salort-Campana, Emmanuelle, Sole, Guilhem, Solera, Luca, Souto Lopes, Suzara, Specht, Sabine, Statland, Jeffrey, Swenson, Andrea, Tan, Chong Yew, Tizon, Sónia, van der Beek, N A M E, van Kooten, Harmke A., Wencel, Marie, Wenninger, Stephan, Zagnoli, Fabien

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Caspase inhibitors can counteract inflammasome activation and caspase-1 mediated fibrosis in Fabry disease von Kasaci, Neil, Dao, Julia, Noll, Lauren, Friedman, Andrew, Ivanova, Margarita, Goker-Alpan, Ozlem

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Lyso-Gb1 acts as a danger-associated molecular pattern (DAMP) to induce cell death and autonomous disease progression, indicating the role of additional therapeutics in Gaucher dis... von Kasaci, Neil, Dao, Julia, Zhang, Claire, Loynab, Neala, Noll, Lauren, Friedman, Andrew, Ivanova, Margarita, Goker-Alpan, Ozlem

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Wnt signaling pathway inhibitor, sclerostin, correlates with bone pain and bone marrow infiltration in patients with Gaucher disease von Dao, Julia, Noll, Lauren, Friedman, Andrew, Goker-Alpan, Ozlem, Ivanova, Margarita

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The expression and secretion profile of TRAP5 isomers correlate with bone involvement in Gaucher disease von Kasaci, Neil, Dao, Julia, Noll, Lauren, Friedman, Andrew, Goker-Alpan, Ozlem, Ivanova, Margarita

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Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial von Kishnani, Priya S, Diaz-Manera, Jordi, Toscano, Antonio, Clemens, Paula R, Ladha, Shafeeq, Berger, Kenneth I, Kushlaf, Hani, Straub, Volker, Carvalho, Gerson, Mozaffar, Tahseen, Roberts, Mark, Attarian, Shahram, Chien, Yin-Hsiu, Choi, Young-Chul, Day, John W, Erdem-Ozdamar, Sevim, Illarioshkin, Sergey, Goker-Alpan, Ozlem, Kostera-Pruszczyk, Anna, van der Ploeg, Ans T, An Haack, Kristina, Huynh-Ba, Olivier, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, Dimachkie, Mazen M, Schoser, Benedikt

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TRAP5b and RANKL/OPG Predict Bone Pathology in Patients with Gaucher Disease von Ivanova, Margarita, Dao, Julia, Noll, Lauren, Fikry, Jacqueline, Goker-Alpan, Ozlem

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Testing the waters: IACUC issues associated with fish von Smith, Stephen A, Noll, Laura E

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33rd international mammalian genome conference: meeting highlights von Tracey, Lauren J., Noll, Kelsey E., Montagutelli, Xavier

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SDHB knockout and succinate accumulation are insufficient for tumorigenesis but dual SDHB/NF1 loss yields SDHx-like pheochromocytomas von Armstrong, Neali, Storey, Claire M., Noll, Sarah E., Margulis, Katherine, Soe, Myat Han, Xu, Haixia, Yeh, Benjamin, Fishbein, Lauren, Kebebew, Electron, Howitt, Brooke E., Zare, Richard N., Sage, Julien, Annes, Justin P.

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1014 POLYPHARMACY IS ASSOCIATED WITH OVERT HEPATIC ENCEPHALOPATHY IN PATIENTS WITH CIRRHOSIS von Montrose, Jonathan, Campbell, Noll, Desai, Archita P., Nephew, Lauren, Patidar, Kavish R., Ghabril, Marwan S., Qiu, Yingjie, Orman, Eric

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Tu1501 ANTICHOLINERGIC MEDICATIONS MAY INCREASE THE RISK OF OVERT HEPATIC ENCEPHALOPATHY IN PATIENTS WITH CIRRHOSIS von Montrose, Jonathan, Campbell, Noll, Desai, Archita P., Nephew, Lauren, Patidar, Kavish R., Ghabril, Marwan S., Qiu, Yingjie, Orman, Eric

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Measuring LEND Core Competencies Using Trainee Follow-Up Surveys von Bishop, Lauren, McLean, Kiley J., Harris, Anne Bradford, Rabidoux, Paula C., Laughlin, Sarah F., Noll, Robert B.

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Virtual Delivery of A School-Based Child Sexual Abuse Prevention Program: A Pilot Study von Guastaferro, Kate, Holloway, Jacqueline L., Trudeau, Jessica, Lipson, Lauren B., Sunshine, S., Noll, Jennie G., Pulido, Mary L.

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Confusion assessment method accurately screens for hepatic encephalopathy and predicts short-term mortality in hospitalized patients with cirrhosis von Desai, Archita P., Gandhi, Devika, Xu, Chenjia, Ghabril, Marwan, Nephew, Lauren, Patidar, Kavish R., Campbell, Noll L., Chalasani, Naga, Boustani, Malaz, Orman, Eric S.

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A model to evaluate interprofessional training effectiveness: feasibility and five-year outcomes of a multi-site prospective cohort study von Bishop, Lauren, Harris, Anne Bradford, Rabidoux, Paula C., Laughlin, Sarah F., McLean, Kiley J., Noll, Robert B.

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The Human Affectome von Alia-Klein, Nelly, Cromwell, Howard C., Eslinger, Paul J., Kemp, Andrew H., Raber, Jacob, Silton, Rebecca L., Williams, Justin H.G., Abe, Nobuhito, Aghajani, Moji, Alexander, Rebecca, Anders, Silke, Arias, Juan A., Arzy, Shahar, Aue, Tatjana, Baez, Sandra, Banta, Marlissa C., Barrett, Karen Caplovitz, Booij, Linda, Bookwala, Jamila, Boutros, Sydney Weber, Bruno, Antonio, Busatto, Geraldo, Bylsma, Lauren M., Caldwell-Harris, Catherine, Cherbuin, Nicolas, Croote, Denise E., Denson, Thomas F., Depue, Brendan, Derntl, Birgit, Drach-Zahavy, Anat, Ellingsen, Dan-Mikael, Fairfield, Beth, Ferdenzi, Camille, Friedman, Bruce H., de Gelder, Beatrice, Gendolla, Guido H.E., Gooding, Anne Elizabeth Kotynski, Gosseries, Olivia, Hanson, Jamie L., Jovanovic, Tanja, Kahrilas, Ian J., Katsumi, Yuta, Kensinger, Elizabeth, Koster, Ernst H.W., Kozlowska, Kasia, Krach, Sören, Kret, Mariska E., Kusi-Mensah, Kwabena, Ladouceur, Cecile D., Liddell, Belinda J., Lidhar, Navdeep K., Lowry, Christopher A., Mariotti, Veronica, Martin, Loren J., Marusak, Hilary A., Mayer, Annalina V., Merner, Amanda R., Morrison, Robert G., Moore, Matthew, Mueller, Sven C., Murphy, Nora A., Muscatello, Maria Rosaria Anna, Musser, Erica D., Norrholm, Seth Davin, Northoff, Georg, Nusslock, Robin, Ortner, Catherine, Owolabi, Mayowa, Padulo, Caterina, Palumbo, Sara, Pellegrini, Silvia, Peltonen, Kirsi, Penninx, Brenda W.J.H., Pinna, Graziano, Rabinak, Christine, Helene Richter, S., Richter, Thalia, Riva, Giuseppe, Robinson, Jennifer L., Rosa, Pedro, Schroeter, Matthias L., Shiban, Youssef, Siddharthan, Advaith, Siedlecka, Ewa, Smith, Robert C., Spangler, Derek P., Stern, Emily R., Styliadis, Charis, Swain, James E., Van den Stock, Jan, vander Kooij, Michael A., van Overveld, Mark, VanElzakker, Michael B., Ventura-Bort, Carlos, Verona, Edelyn, Wang, Yi, Weymar, Mathias, Williams, Claire, Zahn, Roland, Lowe, Leroy

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