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1. Towards the international interoperability of clinical research networks for rare diseases: recommendations from the IRDiRC Task Force

Author

Nabbout, Rima, Zanello, Galliano, Baker, Dixie, Black, Lora, Brambilla, Isabella, Buske, Orion J., Conklin, Laurie S., Davies, Elin Haf, Julkowska, Daria, Kim, Yeonju, Klopstock, Thomas, Nakamura, Harumasa, Nielsen, Kim G., Pariser, Anne R., Pastor, Jose Carlos, Scarpa, Maurizio, Smith, Maureen, Taruscio, Domenica, and Groft, Stephen

Published
2023
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4. Therapeutics for Inflammatory Bowel Diseases in Children and Adolescents: A Focus on Biologics and an Individualized Treatment Paradigm

Author

Batra, Suruchi, Conklin, Laurie S., Barrett, James E., Editor-in-Chief, Flockerzi, Veit, Editorial Board Member, Frohman, Michael A., Editorial Board Member, Geppetti, Pierangelo, Editorial Board Member, Hofmann, Franz B., Editorial Board Member, Michel, Martin C., Editorial Board Member, Page, Clive P., Editorial Board Member, Wang, KeWei, Editorial Board Member, Kiess, Wieland, editor, Schwab, Matthias, editor, and van den Anker, Johannes, editor

Published
2020
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5. Outcomes in Adult Inflammatory Bowel Disease Clinical Trials: Assessment of Similarity Among Participants with Adolescent-onset and Adult-onset Disease.

Author

Rosh, Joel R, Turner, Dan, Hyams, Jeffrey S, Dubinsky, Marla, Griffiths, Anne M, Cohen, Stanley A, Lo, Kim Hung, Kim, Lilianne, Volger, Sheri, Zhang, Renping, Strauss, Richard, and Conklin, Laurie S

Abstract

Background and Aims Most paediatric inflammatory bowel disease [IBD] studies are performed after medications are approved in adults, and the majority of participants in these studies are adolescents. We hypothesised that adolescent-onset IBD is not fundamentally different from adult-onset IBD. If this is correct, the value of delaying access to novel drugs in adolescents becomes questioned. Methods Data from 11 randomised, double-blind, placebo-controlled, adult Phases 2 and 3 trials of four biologics were analysed. Participants were categorised as having adolescent- or adult-onset disease [diagnosed 12 to <18, or ≥18 years]. Multivariable modelling explored the association between age at diagnosis and response to treatment, after adjustment for disease duration, extent, and severity at baseline. Data from dose arms were pooled to evaluate similarity of therapeutic response between adolescent- and adult-onset IBD within the same trial [not between doses or across trials]. Ratios of odds ratios [ORs] between the two groups were evaluated. Results Data from 6283 study participants (2575 with Crohn's disease [CD], 3708 with ulcerative colitis [UC]) were evaluated. Of 2575 study participants with CD, 325 were 12–<18 years old at diagnosis; 836 participants [32.4%] received placebo. Of 3708 participants with UC, 221 were 12–<18 years old at diagnosis; 1212 [33%] were receiving placebo. The majority of the ratios of ORs were within 2-fold, suggesting that responses in adolescent- and adult-onset participants are generally similar. Conclusion Data presented lend support for extrapolating efficacy of biologics from adults to adolescents with IBD, which would facilitate earlier labelling and patient access. [ABSTRACT FROM AUTHOR]

Published
2024
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10. Towards the international interoperability of clinical research networks for rare diseases:recommendations from the IRDiRC Task Force

Author

Nabbout, Rima, Zanello, Galliano, Baker, Dixie, Black, Lora, Brambilla, Isabella, Buske, Orion J., Conklin, Laurie S., Davies, Elin Haf, Julkowska, Daria, Kim, Yeonju, Klopstock, Thomas, Nakamura, Harumasa, Nielsen, Kim G., Pariser, Anne R., Pastor, Jose Carlos, Scarpa, Maurizio, Smith, Maureen, Taruscio, Domenica, Groft, Stephen, Nabbout, Rima, Zanello, Galliano, Baker, Dixie, Black, Lora, Brambilla, Isabella, Buske, Orion J., Conklin, Laurie S., Davies, Elin Haf, Julkowska, Daria, Kim, Yeonju, Klopstock, Thomas, Nakamura, Harumasa, Nielsen, Kim G., Pariser, Anne R., Pastor, Jose Carlos, Scarpa, Maurizio, Smith, Maureen, Taruscio, Domenica, and Groft, Stephen

Abstract

Background: Many patients with rare diseases are still lacking a timely diagnosis and approved therapies for their condition despite the tremendous efforts of the research community, biopharmaceutical, medical device industries, and patient support groups. The development of clinical research networks for rare diseases offers a tremendous opportunity for patients and multi-disciplinary teams to collaborate, share expertise, gain better understanding on specific rare diseases, and accelerate clinical research and innovation. Clinical Research Networks have been developed at a national or continental level, but global collaborative efforts to connect them are still lacking. The International Rare Diseases Research Consortium set a Task Force on Clinical Research Networks for Rare Diseases with the objective to analyse the structure and attributes of these networks and to identify the barriers and needs preventing their international collaboration. The Task Force created a survey and sent it to pre-identified clinical research networks located worldwide. Results: A total of 34 responses were received. The survey analysis demonstrated that clinical research networks are diverse in their membership composition and emphasize community partnerships including patient groups, health care providers and researchers. The sustainability of the networks is mostly supported by public funding. Activities and research carried out at the networks span the research continuum from basic to clinical to translational research studies. Key elements and infrastructures conducive to collaboration are well adopted by the networks, but barriers to international interoperability are clearly identified. These hurdles can be grouped into five categories: funding limitation; lack of harmonization in regulatory and contracting process; need for common tools and data standards; need for a governance framework and coordination structures; and lack of awareness and robust interactions between netw

Published
2023

11. Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

Author

Hoffman, Eric P., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Smith, Edward C., Castro, Diana, Mah, Jean K., McDonald, Craig M., Kuntz, Nancy L., Finkel, Richard S., Guglieri, Michela, Bushby, Katharine, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Smith, Andrea L., Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Siener, Catherine, Jaros, Mark, Shale, Phil, McCall, John M., Nagaraju, Kanneboyina, van den Anker, John, Conklin, Laurie S., Cnaan, Avital, Gordish-Dressman, Heather, Damsker, Jesse M., and Clemens, Paula R.

Published
2019
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12. Acute Hepatitis

Author

Badalyan, Vahe, primary, Conklin, Laurie S., additional, and Snyder, John D., additional

Published
2018
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13. Contributors

Author

Abzug, Mark J., primary, Adderson, Elisabeth E., additional, Agwu, Allison L., additional, Alby, Kevin, additional, Aldrovandi, Grace M., additional, Allen, Upton D., additional, Alvarez-Hernández, Gerardo, additional, Ampofo, Krow, additional, Anderson, Evan J., additional, Anderson, Margot, additional, Antonara, Stella, additional, Ardura, Monica I., additional, Arguin, Paul M., additional, Arnold, John C., additional, Aronson, Naomi E., additional, Arvin, Ann M., additional, Ashkenazi, Shai, additional, Asturias, Edwin J., additional, Badalyan, Vahe, additional, Baker, Carol J., additional, Balakrishnan, Karthik, additional, Barros, Brittany S., additional, Barson, William J., additional, Bausch, Daniel G., additional, Bechtel, Kirsten, additional, Benjamin, Daniel K., additional, Berman, David M., additional, Blanco, David A., additional, Bloch, Karen C., additional, Blythe, Margaret J., additional, Bocchini, Joseph A., additional, Bowen, Anna, additional, Bowie, William R., additional, Boyce, Thomas G., additional, Bradley, John S., additional, Brady, Michael T., additional, Bratcher, Denise F., additional, Braverman, Paula K., additional, Bresee, Joseph, additional, Brook, Itzhak, additional, Brown, Kevin E., additional, Bryant, Kristina, additional, Buescher, E. Stephen, additional, Burns, Jane L., additional, Byington, Carrie L., additional, Camacho-Gonzalez, Andres F., additional, Cantey, Paul, additional, Carter, Bryan D., additional, Caserta, Mary T., additional, Castagnini, Luis A., additional, Cerini, Chiara, additional, Chadwick, Ellen Gould, additional, Chiang, Silvia S., additional, Christenson, John C., additional, Coffin, Susan E., additional, Collier, Melissa G., additional, Collins, Jennifer P., additional, Conklin, Laurie S., additional, Connelly, Beverly L., additional, Contopoulos-Ioannidis, Despina, additional, Conway, James H., additional, Cortese, Margaret M., additional, Cox, Elaine G., additional, Creech, C. Buddy, additional, Crews, Jonathan D., additional, Cunningham, Dennis J., additional, Curtis, Nigel, additional, Dailey, Natalie J.M., additional, Danziger-Isakov, Lara A., additional, Darville, Toni, additional, Dasch, Gregory A., additional, Daskalaki, Irini, additional, Daum, Robert S., additional, Davenport, Michael, additional, Davies, H. Dele, additional, Dawood, Fatimah S., additional, Day, J. Christopher, additional, de la Morena, Maite, additional, Demmler-Harrison, Gail J., additional, DeMuri, Gregory P., additional, Despommier, Dickson D., additional, Diefenbach, Karen A., additional, Edwards, Kathryn M., additional, Edwards, Morven S., additional, Eichenfield, Lawrence F., additional, Elston, Dirk M., additional, Emerson, Beth, additional, Ephros, Moshe, additional, Erdem, Guliz, additional, Eremeeva, Marina E., additional, Ericson, Jessica E., additional, Esposito, Douglas H., additional, Farley, Monica M., additional, Feingold, Anat R., additional, Feja, Kristina N., additional, Finn, Adam, additional, Fischer, Marc, additional, Flynn, Patricia M., additional, Fox, LeAnne M., additional, Frank, Michael M., additional, Fredrick, Douglas R., additional, Frenck, Robert W., additional, Friedlander, Sheila Fallon, additional, Gans, Hayley A., additional, Gauthier, Gregory M., additional, Gerber, Jeffrey S., additional, Gigliotti, Francis, additional, Gilger, Mark A., additional, Glaser, Carol A., additional, Goddard, Amanda F., additional, Gold, Benjamin D., additional, Gould, Jane M., additional, Green, Michael, additional, Greenberg, David, additional, Greywal, Tanya, additional, Griffin, Daniel, additional, Griffin, Patricia M., additional, Grom, Alexei A., additional, Gutierrez, Kathleen, additional, Gutman, Julie, additional, Guzman-Cottrill, Judith A., additional, Hall, Aron J., additional, Han, Jin-Young, additional, Harper, Marvin B., additional, Harris, Julie R., additional, Harrison, Christopher J., additional, Haslam, David B., additional, Hawkes, Sarah J., additional, Hendley, J. Owen, additional, Henry, Marion C.W., additional, Hilinski, Joseph A., additional, Hills, Susan L., additional, Holmberg, Scott D., additional, Holtzman, Deborah, additional, Hong, David K., additional, Hotez, Peter J., additional, Hsu, Katherine K., additional, Hunstad, David A., additional, Hwang, Loris Y., additional, Jackson, Mary Anne, additional, Jacobs, Richard F., additional, Jhaveri, Ravi, additional, Jirků-Pomajbíková, Kateřina, additional, Jones, Jeffrey L., additional, Karki, Mahima, additional, Karlowicz, M. Gary, additional, Katz, Ben Z., additional, Kaur, Ishminder, additional, Kersh, Gilbert J., additional, Keystone, Jay S., additional, Khan, Muhammad Ali, additional, Kimberlin, David W., additional, Kleiman, Martin B., additional, Klein, Bruce S., additional, Klontz, Karl, additional, Knust, Barbara, additional, Koh, Andrew Y., additional, Korgenski, E. Kent, additional, Krogstad, Paul, additional, Kutty, Preeta Krishnan, additional, Lauren, Christine T., additional, Lawrence, Hillary S., additional, Leber, Amy, additional, Lee, Grace M., additional, Leibovitz, Eugene, additional, Leshem, Eyal, additional, Levasseur, Stéphanie, additional, Lewis, David B., additional, Livingston, Robyn A., additional, Llata, Eloisa, additional, Long, Sarah S., additional, Lopman, Ben A., additional, Lucero, Yalda C., additional, Luján-Zilbermann, Jorge, additional, Luzuriaga, Katherine, additional, MacDonald, Noni E., additional, Maldonado, Yvonne A., additional, Manaloor, John, additional, Mani, Chitra S., additional, Manthiram, Kalpana, additional, Marshall, Gary S., additional, Martin, Stacey W., additional, Matanock, Almea, additional, Matiz, Catalina, additional, Mawle, Alison C., additional, Mazzulli, Tony, additional, McGann, Kathleen A., additional, McIntosh, Kenneth, additional, McNamara, Lucy A., additional, Meir, Michal, additional, Meislich, Debrah, additional, Meissner, H. Cody, additional, Meites, Elissa, additional, Mejías, Asunción, additional, Mertsola, Jussi, additional, Messacar, Kevin, additional, Mhaissen, Mohammed Nael, additional, Michaels, Marian G., additional, Miller, Melissa B., additional, Mintz, Eric D., additional, Modlin, John F., additional, Mohan, Parvathi, additional, Montgomery, Susan P., additional, Montoya, José G., additional, Moro, Pedro L., additional, Moscicki, Anna-Barbara, additional, Moss, R. Lawrence, additional, Myers, Angela L., additional, Nadel, Simon, additional, Neely, Michael N., additional, Neil, Karen P., additional, Nelson, Joanna, additional, Nelson, Noele P., additional, Nicholson, William L., additional, Nizet, Victor, additional, Nopper, Amy Jo, additional, Ochoa, Theresa J., additional, Orenstein, Walter A., additional, O'Ryan, Miguel, additional, Paddock, Christopher D., additional, Pall, Harpreet, additional, Panuganti, Suresh Kumar, additional, Pappas, Diane E., additional, Pass, Robert F., additional, Patterson, Thomas F., additional, Patton, Monica E., additional, Pelton, Stephen I., additional, Petersen, Brett W., additional, Pickering, Larry K., additional, Pinninti, Swetha, additional, Planet, Paul J., additional, Pollard, Andrew J., additional, Posfay-Barbe, Klara M., additional, Poulsen, Casper S., additional, Poutanen, Susan M., additional, Powers, Ann M., additional, Prober, Charles G., additional, Ramilo, Octavio, additional, Rangel, Shawn J., additional, Rao, Suchitra, additional, Rawstron, Sarah A., additional, Read, Jennifer S., additional, Reed, Michael D., additional, Relich, Ryan F., additional, Reller, Megan E., additional, Rellosa, Neil, additional, Rempe, Katherine A., additional, Reyes, Melissa A., additional, Rice-Townsend, Samuel E., additional, Richards, Frank O., additional, Romero, José R., additional, Rosen, David A., additional, Rostad, Christina A., additional, Rours, G. Ingrid J.G., additional, Routh, Janell A., additional, Rowley, Anne H., additional, Rubin, Lorry G., additional, Ryan, Edward T., additional, Saiman, Lisa, additional, Sammons, Julia S., additional, Sass, Laura, additional, Sauberan, Jason B., additional, Schillie, Sarah, additional, Schulert, Grant S., additional, Schuster, Jennifer E., additional, Schwartz, Kevin L., additional, Sederdahl, Bethany K., additional, Serpa, Jose A., additional, Shah, Kara N., additional, Shah, Samir S., additional, Shane, Andi L., additional, Shapiro, Eugene D., additional, Shaw, Jana, additional, Shetty, Avinash K., additional, Dairiki, Linda M., additional, Siberry, George Kelly, additional, Siegel, Jane D., additional, Siegel, Robert David, additional, Simonsen, Kari A., additional, Singh, Nalini, additional, Singh, Upinder, additional, Smith, P. Brian, additional, Snyder, John D., additional, Song, Eunkyung, additional, Sorrell, Jennifer L., additional, Souder, Emily, additional, St. Geme, Joseph W., additional, Staat, Mary Allen, additional, Staples, J. Erin, additional, Starke, Jeffrey R., additional, Steinbach, William J., additional, Stensvold, Christen R., additional, Stoner, Bradley P., additional, Strikas, Raymond A., additional, Strober, Jonathan B., additional, Sue, Paul K., additional, Sutton, Deanna A., additional, Swanson, Douglas, additional, Tate, Jacqueline E., additional, Tebruegge, Marc, additional, Teshale, Eyasu H., additional, Thompson, Amelia B., additional, Thompson, George R., additional, Thompson-Stone, Robert, additional, Thomson, Richard B., additional, Thorell, Emily A., additional, Tobin, Nicole H., additional, Toltzis, Philip, additional, Treat, James, additional, Troy, Stephanie B., additional, Van, Russell B., additional, Vaz, Louise Elaine, additional, Vodzak, Jennifer, additional, Wald, Ellen R., additional, Wallihan, Rebecca, additional, Wangu, Zoon, additional, Washam, Matthew, additional, Watson, Joshua R., additional, Wattier, Rachel L., additional, Weinberg, Geoffrey A., additional, White, A. Clinton, additional, Wiesenfeld, Harold C., additional, Williams, John V., additional, Willoughby, Rodney E., additional, Wingerter, Sarah L., additional, Wittler, Robert R., additional, Wong, Karen K., additional, Workowski, Kimberly A., additional, Wright, Terry W., additional, Yagupsky, Pablo, additional, Yen, Catherine, additional, Yi, Jumi, additional, Yoder, Jonathan S., additional, Young, Edward J., additional, Zaenglein, Andrea L., additional, and Zimmerman, Kanecia, additional

Published
2018
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16. Using multiple imputation of real-world data to estimate clinical remission in pediatric inflammatory bowel disease

Author

Zhang, Nanhua, primary, Liu, Chunyan, additional, Steiner, Steven J, additional, Colletti, Richard B, additional, Baldassano, Robert, additional, Chen, Shiran, additional, Cohen, Stanley, additional, Kappelman, Michael D, additional, Saeed, Shehzad, additional, Conklin, Laurie S, additional, Strauss, Richard, additional, Volger, Sheri, additional, King, Eileen, additional, and Lo, Kim Hung, additional

Published
2023
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17. Innovations in Pediatric Therapeutics Development: Principles for the Use of Bridging Biomarkers in Pediatric Extrapolation

Author

Fleming, Thomas R., primary, Garnett, Christine E., additional, Conklin, Laurie S., additional, Corriol-Rohou, Solange, additional, Hariharan, Sudharshan, additional, Hsu, Daphne, additional, Mueller-Velten, Guenther, additional, Mulugeta, Yeruk, additional, Portman, Ronald, additional, Rothmann, Mark D., additional, Stockbridge, Norman L., additional, Wandel, Simon, additional, Zhang, Jialu, additional, and Yao, Lynne, additional

Published
2022
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18. Validating the Simplified Endoscopic Mucosal Assessment for Crohn’s Disease: A Novel Method for Assessing Disease Activity

Author

Adler, Jeremy, primary, Colletti, Richard B, additional, Noonan, Lenore, additional, Berzin, Tyler M, additional, Cheifetz, Adam S, additional, Conklin, Laurie S, additional, Hoops, Timothy C, additional, Huang, Christopher S, additional, Lewis, Blair, additional, Mishkin, Daniel S, additional, Hung Lo, Kim, additional, Xiao, Yongling, additional, and Volger, Sheri, additional

Published
2022
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19. TECHNIQUES, TECHNOLOGY & ANALYSIS: POSITION PAPER: The potential role of optical biopsy in the study and diagnosis of environmental enteric dysfunction

Author

Thompson, Alex J., Hughes, Michael, Anastasova, Salzitsa, Conklin, Laurie S., Thomas, Tudor, Leggett, Cadman, Faubion, William A., Miller, Thomas J., Delaney, Peter, Lacombe, François, Loiseau, Sacha, Meining, Alexander, Richards-Kortum, Rebecca, Tearney, Guillermo J., Kelly, Paul, and Yang, Guang-Zhong

Published
2017
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22. Pediatric Extrapolation of Adult Efficacy to Children Is Critical for Efficient and Successful Drug Development

Author

Mulberg, Andrew E., primary, Conklin, Laurie S., additional, Croft, Nicholas M., additional, Parikh, Anushree, additional, Bousvaros, Athos, additional, DeZoeten, Edwin, additional, Dubinsky, Marla, additional, Martin Carpi, Francisco Javier, additional, Escher, Hankje, additional, Vesely, Richard, additional, Ollivier, Cecile, additional, Crandall, Wallace, additional, and Hovinga, Collin, additional

Published
2022
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23. Enhanced TH17 Responses in Patients with IL10 Receptor Deficiency and Infantile-onset IBD

Author

Shouval, Dror S., Konnikova, Liza, Griffith, Alexandra E., Wall, Sarah M., Biswas, Amlan, Werner, Lael, Nunberg, Moran, Kammermeier, Jochen, Goettel, Jeremy A., Anand, Rajsavi, Chen, Hannah, Weiss, Batia, Li, Jian, Loizides, Anthony, Yerushalmi, Baruch, Yanagi, Tadahiro, Beier, Rita, Conklin, Laurie S., Ebens, Christen L., Santos, Fernanda G. M. S., Sherlock, Mary, Goldsmith, Jeffery D., Kotlarz, Daniel, Glover, Sarah C., Shah, Neil, Bousvaros, Athos, Uhlig, Holm H., Muise, Aleixo M., Klein, Christoph, and Snapper, Scott B.

Published
2017
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27. Interleukin-10 Receptor Signaling in Innate Immune Cells Regulates Mucosal Immune Tolerance and Anti-Inflammatory Macrophage Function

Author

Shouval, Dror S., Biswas, Amlan, Goettel, Jeremy A., McCann, Katelyn, Conaway, Evan, Redhu, Naresh S., Mascanfroni, Ivan D., Al Adham, Ziad, Lavoie, Sydney, Ibourk, Mouna, Nguyen, Deanna D., Samsom, Janneke N., Escher, Johanna C., Somech, Raz, Weiss, Batia, Beier, Rita, Conklin, Laurie S., Ebens, Christen L., Santos, Fernanda G.M.S., Ferreira, Alexandre R., Sherlock, Mary, Bhan, Atul K., Müller, Werner, Mora, Rodrigo J., Quintana, Francisco J., Klein, Christoph, Muise, Aleixo M., Horwitz, Bruce H., and Snapper, Scott B.

Published
2014
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30. Contributors

Author

Adderson, Elisabeth E., primary, Agarwal, Aarti, additional, Aldrovandi, Grace M., additional, Allen, Upton D., additional, Amieva, Manuel R., additional, Ampofo, Krow, additional, Anderson, Alicia D., additional, Anderson, Margot, additional, Arguin, Paul M., additional, Arnold, John C., additional, Arvin, Ann M., additional, Ashkenazi, Shai, additional, Baker, Carol J., additional, Barson, William J., additional, Bausch, Daniel G., additional, Bechtel, Kirsten, additional, Benjamin, Daniel K., additional, Berkowitz, Frank E., additional, Blythe, Margaret J., additional, Bocchini, Joseph A., additional, Boeckh, Michael, additional, Bowen, Anna, additional, Bowie, William R., additional, Boyce, Thomas G., additional, Bradley, John S., additional, Brady, Michael T., additional, Bratcher, Denise F., additional, Braverman, Paula K., additional, Hall, Caroline Breese, additional, Bresee, Joseph S., additional, Brook, Itzhak, additional, Bryant, Kristina, additional, Buescher, E. Stephen, additional, Burns, Jane L., additional, Burstein, Gale R., additional, Byington, Carrie L., additional, Byrd, Kathy K., additional, Cappello, Michael, additional, Carter, Bryan D., additional, Cartwright, Emily J., additional, Caserta, Mary T., additional, Cerini, Chiara, additional, Chadwick, Ellen Gould, additional, Cheesebrough, Beth, additional, Chesney, P. Joan, additional, Christenson, John C., additional, Cleary, Thomas G., additional, Coffin, Susan E., additional, Conklin, Laura M., additional, Conklin, Laurie S., additional, Connelly, Beverly L., additional, Contopoulos-Ioannidis, Despina, additional, Conway, James H., additional, Cortese, Margaret M., additional, Cotten, C. Michael, additional, Cox, Elaine, additional, Crockett, Maryanne E., additional, Crowe, James E., additional, Curtis, Nigel, additional, Cunningham, Dennis J., additional, Shortliffe, Linda Marie Dairiki, additional, Darville, Toni, additional, Dasch, Gregory A., additional, Daskalaki, Irini, additional, Daum, Robert S., additional, Dawood, Fatimah S., additional, Demmler, Gail J., additional, Despommier, Dickson D., additional, Diefenbach, Karen A., additional, Dvorak, Christopher C., additional, Edwards, Kathryn M., additional, Edwards, Morven S., additional, Eichenfield, Lawrence F., additional, Elston, Dirk M., additional, Englund, Janet A., additional, Erard, Veronique, additional, Eremeeva, Marina E., additional, Feingold, Anat R., additional, Finn, Adam, additional, Fiore, Anthony E., additional, Fischer, Marc, additional, Fitch, Sarah J., additional, Flynn, Patricia M., additional, Fox, LeAnne M., additional, Frank, Michael M., additional, Fredrick, Douglas R., additional, Friedlander, Sheila Fallon, additional, Gans, Hayley A., additional, Garcia, Carla G., additional, Garzon, Maria C., additional, Gerber, Jeffrey S., additional, Geschwind, Michael D., additional, Gieraltowski, Laura B., additional, Gigliotti, Francis, additional, Gilligan, Peter H., additional, Glaser, Carol, additional, Gold, Benjamin D., additional, Goldstein, Brahm, additional, Gould, Jane M., additional, Green, Michael, additional, Greenberg, David, additional, Griffin, Patricia M., additional, Grom, Alexei A., additional, Gutierrez, Kathleen, additional, Guzman-Cottrill, Judith A., additional, Hall, Aron J., additional, Harper, Marvin B., additional, Harrison, Christopher J., additional, Haslam, David B., additional, Hawkes, Sarah J., additional, Hayes, Edward B., additional, Hazra, Rohan, additional, Heilig, Sara Jane, additional, Hendley, J. Owen, additional, Henry, Marion C.W., additional, Hilinski, Joseph A., additional, Holmberg, Scott D., additional, Holtzman, Deborah, additional, Hotez, Peter J., additional, Hsu, Katherine K., additional, Hu, Dale J., additional, Hwang, Loris Y., additional, Hyun, David Y., additional, Jackson, Mary Anne, additional, Jacobs, Richard F., additional, Jones, Jeffrey L., additional, Kamili, Saleem, additional, Karlowicz, M. Gary, additional, Katz, Ben Z., additional, Kersh, Gilbert J., additional, Kester, Laura M., additional, Keystone, Jay S., additional, Kimberlin, David W., additional, Kleiman, Martin B., additional, Kline, Mark W., additional, Koh, Andrew Y., additional, Konstantopoulos, Andreas, additional, Koranyi, Katalin I., additional, Korgenski, E. Kent, additional, Kroger, Andrew T., additional, Krogstad, Paul, additional, Lauren, Christine T., additional, Lawrence, Hillary S., additional, Leibovitz, Eugene, additional, Levasseur, Stéphanie, additional, Lewis, David B., additional, Lieberman, Jay M., additional, Liu, Jen-Jane, additional, Livingston, Robyn A., additional, Llata, Eloisa, additional, Loharikar, Anagha R., additional, Long, Sarah S., additional, Lopman, Ben A., additional, Lorber, Bennett, additional, Low, Donald E., additional, Lucero, Yalda C., additional, Luján-Zilbermann, Jorge, additional, Luzuriaga, Katherine, additional, MacDonald, Noni E., additional, MacNeil, Adam, additional, Maldonado, Yvonne A., additional, Mani, Chitra S., additional, Marcon, Mario J., additional, Marshall, Gary S., additional, Martin, Stacey W., additional, Matiz, Catalina, additional, Mawle, Alison C., additional, Mazzulli, Tony, additional, McCracken, George H., additional, McDonald, Matthew B., additional, McGregor, Robert S., additional, McIntosh, Kenneth, additional, McMorrow, Meredith, additional, McNeil, Candice, additional, McQuiston, Jennifer H., additional, Meislich, Debrah, additional, Meissner, H. Cody, additional, Mejías, Asunción, additional, Menon, Manoj P., additional, Mertsola, Jussi, additional, Michaels, Marian G., additional, Miller, Melissa B., additional, Mintz, Eric D., additional, Modlin, John F., additional, Mohan, Parvathi, additional, Montgomery, Susan P., additional, Montoya, Jose G., additional, Moore, Zack S., additional, de la Morena, Maite, additional, Moro, Pedro L., additional, Moscicki, Anna-Barbara, additional, Moss, R. Lawrence, additional, Murphy, Trudy V., additional, Murray, Dennis L., additional, Myers, Angela L., additional, Nadel, Simon, additional, Nataro, James P., additional, Neely, Michael N., additional, Nicholson, William L., additional, Nizet, Victor, additional, Nopper, Amy Jo, additional, Norrby-Teglund, Anna, additional, Ochoa, Theresa J., additional, O’Ryan, Miguel, additional, Orenstein, Walter A., additional, Paddock, Christopher D., additional, Pappas, Diane E., additional, Pass, Robert F., additional, Patterson, Thomas F., additional, Pelton, Stephen I., additional, Pickering, Larry K., additional, Piggott, Caroline Diane Sarah, additional, Pizzo, Philip A., additional, Pollard, Andrew J., additional, Posfay-Barbe, Klara M., additional, Poutanen, Susan M., additional, Powell, Dwight A., additional, Prince, Alice S., additional, Prober, Charles G., additional, Ramilo, Octavio, additional, Rangel, Shawn J., additional, Rawstron, Sarah A., additional, Read, Jennifer S., additional, Reed, Michael D., additional, Regan, Joanna J., additional, Reller, Megan E., additional, Reyes, Melissa A., additional, Rice, Peter A., additional, Rice-Townsend, Samuel E., additional, Richards, Frank O., additional, Rodgers, Gail L., additional, Rollin, Pierre E., additional, Romero, José R., additional, Rours, G. Ingrid J.G., additional, Rowley, Anne H., additional, Roy, Sharon L., additional, Rubin, Lorry G., additional, Ruiz-Palacios, Guillermo M., additional, Saiman, Lisa, additional, Sass, Laura, additional, Sauberan, Jason B., additional, Schantz, Peter M., additional, Schneider, Eileen, additional, Schutze, Gordon E., additional, Schwartz, Benjamin, additional, Schwarzwald, Heidi, additional, Shah, Kara N., additional, Shah, Samir S., additional, Shane, Andi L., additional, Shapiro, Craig A., additional, Shapiro, Eugene D., additional, Sharapov, Umid M., additional, Shaw, Jana, additional, Siberry, George Kelly, additional, Siegel, Jane D., additional, Siegel, Robert David, additional, Singh, Nalini, additional, Singh, Upinder, additional, Smith, P. Brian, additional, Snyder, John D., additional, Soper, David E., additional, Staat, Mary Allen, additional, Staples, J. Erin, additional, Starke, Jeffrey R., additional, Steinbach, William J., additional, Stephens, Ina, additional, St. Geme, Joseph W., additional, Stoner, Bradley P., additional, Strober, Jonathan B., additional, Subbarao, Kanta, additional, Sutton, Deanna A., additional, Swanson, Douglas, additional, Takada, Leonel T., additional, Tate, Jacqueline E., additional, Tauxe, Robert V., additional, Tebruegge, Marc, additional, Teshale, Eyasu H., additional, Thompson, George R., additional, Thompson, Herbert A., additional, Thomson, Richard B., additional, Thorell, Emily A., additional, Tohme, Rania A., additional, Tolan, Robert W., additional, Toltzis, Philip, additional, Treat, James, additional, Troy, Stephanie B., additional, Van Dyke, Russell B., additional, Velarde, Jorge J., additional, Vodzak, Jennifer, additional, Wald, Ellen R., additional, Weinberg, Geoffrey A., additional, White, A. Clinton, additional, Widdowson, Marc-Alain, additional, Wiesenfeld, Harold C., additional, Williams, John V., additional, Williams, Roxanne E., additional, Willoughby, Rodney E., additional, Wilson, Craig M., additional, Wingerter, Sarah L., additional, Winkelstein, Jerry A., additional, Workowski, Kimberly A., additional, Wright, Terry W., additional, Yagupsky, Pablo, additional, Yazigi, Nada, additional, Yen, Catherine, additional, Young, Edward J., additional, Zaenglein, Andrea L., additional, and Zaoutis, Theoklis E., additional

Published
2012
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40. Serum biomarkers of glucocorticoid response and safety in anti-neutrophil cytoplasmic antibody-associated vasculitis and juvenile dermatomyositis

Author

Conklin, Laurie S., primary, Merkel, Peter A., additional, Pachman, Lauren M., additional, Parikh, Hemang, additional, Tawalbeh, Shefa, additional, Damsker, Jesse M., additional, Cuthbertson, David D., additional, Morgan, Gabrielle A., additional, Monach, Paul A., additional, Hathout, Yetrib, additional, Nagaraju, Kanneboyina, additional, van den Anker, John, additional, McAlear, Carol A., additional, and Hoffman, Eric P., additional

Published
2018
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42. Position paper: The potential role of optical biopsy in the study and diagnosis of environmental enteric dysfunction

Author

Thompson, Alex J., Hughes, Michael, Anastasova, Salzitsa, Conklin, Laurie S., Thomas, Tudor, Leggett, Cadman, Faubion, William A., Miller, Thomas J., Delaney, Peter, Lacombe, François, Loiseau, Sacha, Meining, Alexander, Richards-Kortum, Rebecca, Tearney, Guillermo J., Kelly, Paul, and Yang, Guang-Zhong

Abstract

Environmental enteric dysfunction (EED) is a disease of the small intestine affecting children and adults in low and middle income countries. Arising as a consequence of repeated infections, gut inflammation results in impaired intestinal absorptive and barrier function, leading to poor nutrient uptake and ultimately to stunting and other developmental limitations. Progress towards new biomarkers and interventions for EED is hampered by the practical and ethical difficulties of cross-validation with the gold standard of biopsy and histology. Optical biopsy techniques — which can provide minimally invasive or noninvasive alternatives to biopsy — could offer other routes to validation and could potentially be used as point-of-care tests among the general population. This Consensus Statement identifies and reviews the most promising candidate optical biopsy technologies for applications in EED, critically assesses them against criteria identified for successful deployment in developing world settings, and proposes further lines of enquiry. Importantly, many of the techniques discussed could also be adapted to monitor the impaired intestinal barrier in other settings such as IBD, autoimmune enteropathies, coeliac disease, graft-versus-host disease, small intestinal transplantation or critical care.

Published
2017

43. Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.

Author

Smith, Edward C., Conklin, Laurie S., Hoffman, Eric P., Clemens, Paula R., Mah, Jean K., Finkel, Richard S., Guglieri, Michela, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Castro, Diana, Kuntz, Nancy L., Kerchner, Laurie, Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Jaros, Mark, Shale, Phil, and Gordish-Dressman, Heather

Subjects
*DUCHENNE muscular dystrophy, *CLINICAL trial registries, *STUNTED growth, *BEHAVIOR, *STAIR climbing, *INVESTIGATIONAL drugs
Abstract

Background: Treatment with corticosteroids is recommended for Duchenne muscular dystrophy (DMD) patients to slow the progression of weakness. However, chronic corticosteroid treatment causes significant morbidities. Vamorolone is a first-in-class anti-inflammatory investigational drug that has shown evidence of efficacy in DMD after 24 weeks of treatment at 2.0 or 6.0 mg/kg/day. Here, open-label efficacy and safety experience of vamorolone was evaluated over a period of 18 months in trial participants with DMD.Methods and Findings: A multicenter, open-label, 24-week trial (VBP15-003) with a 24-month long-term extension (VBP15-LTE) was conducted by the Cooperative International Neuromuscular Research Group (CINRG) and evaluated drug-related effects of vamorolone on motor outcomes and corticosteroid-associated safety concerns. The study was carried out in Canada, US, UK, Australia, Sweden, and Israel, from 2016 to 2019. This report covers the initial 24-week trial and the first 12 months of the VBP15-LTE trial (total treatment period 18 months). DMD trial participants (males, 4 to <7 years at entry) treated with 2.0 or 6.0 mg/kg/day vamorolone for the full 18-month period (n = 23) showed clinical improvement of all motor outcomes from baseline to month 18 (time to stand velocity, p = 0.012 [95% CI 0.010, 0.068 event/second]; run/walk 10 meters velocity, p < 0.001 [95% CI 0.220, 0.491 meters/second]; climb 4 stairs velocity, p = 0.001 [95% CI 0.034, 0.105 event/second]; 6-minute walk test, p = 0.001 [95% CI 31.14, 93.38 meters]; North Star Ambulatory Assessment, p < 0.001 [95% CI 2.702, 6.662 points]). Outcomes in vamorolone-treated DMD patients (n = 46) were compared to group-matched participants in the CINRG Duchenne Natural History Study (corticosteroid-naïve, n = 19; corticosteroid-treated, n = 68) over a similar 18-month period. Time to stand was not significantly different between vamorolone-treated and corticosteroid-naïve participants (p = 0.088; least squares [LS] mean 0.042 [95% CI -0.007, 0.091]), but vamorolone-treated participants showed significant improvement compared to group-matched corticosteroid-naïve participants for run/walk 10 meters velocity (p = 0.003; LS mean 0.286 [95% CI 0.104, 0.469]) and climb 4 stairs velocity (p = 0.027; LS mean 0.059 [95% CI 0.007, 0.111]). The vamorolone-related improvements were similar in magnitude to corticosteroid-related improvements. Corticosteroid-treated participants showed stunting of growth, whereas vamorolone-treated trial participants did not (p < 0.001; LS mean 15.86 [95% CI 8.51, 23.22]). Physician-reported incidences of adverse events (AEs) for Cushingoid appearance, hirsutism, weight gain, and behavior change were less for vamorolone than published incidences for prednisone and deflazacort. Key limitations to the study were the open-label design, and use of external comparators.Conclusions: We observed that vamorolone treatment was associated with improvements in some motor outcomes as compared with corticosteroid-naïve individuals over an 18-month treatment period. We found that fewer physician-reported AEs occurred with vamorolone than have been reported for treatment with prednisone and deflazacort, and that vamorolone treatment did not cause the stunting of growth seen with these corticosteroids. This Phase IIa study provides Class III evidence to support benefit of motor function in young boys with DMD treated with vamorolone 2.0 to 6.0 mg/kg/day, with a favorable safety profile. A Phase III RCT is underway to further investigate safety and efficacy.Trial Registration: Clinical trials were registered at www.clinicaltrials.gov, and the links to each trial are as follows (as provided in manuscript text): VBP15-002 [NCT02760264] VBP15-003 [NCT02760277] VBP15-LTE [NCT03038399]. [ABSTRACT FROM AUTHOR]

Published
2020
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45. Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

Author

Conklin, Laurie S., primary, Damsker, Jesse M., additional, Hoffman, Eric P., additional, Jusko, William J., additional, Mavroudis, Panteleimon D., additional, Schwartz, Benjamin D., additional, Mengle-Gaw, Laurel J., additional, Smith, Edward C., additional, Mah, Jean K., additional, Guglieri, Michela, additional, Nevo, Yoram, additional, Kuntz, Nancy, additional, McDonald, Craig M., additional, Tulinius, Mar, additional, Ryan, Monique M., additional, Webster, Richard, additional, Castro, Diana, additional, Finkel, Richard S., additional, Smith, Andrea L., additional, Morgenroth, Lauren P., additional, Arrieta, Adrienne, additional, Shimony, Maya, additional, Jaros, Mark, additional, Shale, Phil, additional, McCall, John M., additional, Hathout, Yetrib, additional, Nagaraju, Kanneboyina, additional, van den Anker, John, additional, Ward, Leanne M., additional, Ahmet, Alexandra, additional, Cornish, Michaelyn R., additional, and Clemens, Paula R., additional

Published
2018
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46. Pediatric Inflammatory Bowel Disease Clinical Innovations Meeting of the Crohn’s & Colitis Foundation: Charting the Future of Pediatric IBD

Author

Picoraro, Joseph A, primary, Lee, Dale, additional, Heller, Caren A, additional, Weaver, Alandra, additional, Hyams, Jeffrey S, additional, Conklin, Laurie S, additional, Otley, Anthony, additional, Ziring, David, additional, Kugathasan, Subra, additional, Rosh, Joel R, additional, Mulberg, Andrew, additional, Denson, Lee A, additional, Kappelman, Michael D, additional, Grossman, Andrew B, additional, Bousvaros, Athos, additional, and Park, K T, additional

Published
2018
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47. Obesity and Pediatric Drug Development

Author

Vaughns, Janelle D., primary, Conklin, Laurie S., additional, Long, Ying, additional, Zheng, Panli, additional, Faruque, Fahim, additional, Green, Dionna J., additional, den Anker, John N., additional, and Burckart, Gilbert J., additional

Published
2018
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49. The potential role of optical biopsy in the study and diagnosis of environmental enteric dysfunction

Author

Thompson, Alex J., primary, Hughes, Michael, additional, Anastasova, Salzitsa, additional, Conklin, Laurie S., additional, Thomas, Tudor, additional, Leggett, Cadman, additional, Faubion, William A., additional, Miller, Thomas J., additional, Delaney, Peter, additional, Lacombe, François, additional, Loiseau, Sacha, additional, Meining, Alexander, additional, Richards-Kortum, Rebecca, additional, Tearney, Guillermo J., additional, Kelly, Paul, additional, and Yang, Guang-Zhong, additional

Published
2017
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