20 results on '"Patel, Kapilkumar"'
Search Results
2. Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study
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Gibson, Ronald L., Jr., Rowe, Steven M., Lechtzin, Noah, Ahrens, Richard C., McCoy, Karen S., Aitken, Moira, Donaldson, Scott H., McBennett, Kimberly Ann, Pilewski, Joseph M., Billings, Joanne, Milla, Carlos, Rubenstein, Ronald, Rosenbluth, Daniel Brian, Linnemann, Rachel, Powers, Michael R., Fortner, Christopher, Frederick, Carla Anne, Liou, Theodore G., Black, Philip, Wang, Janice, Colombo, John L., Berdella, Maria, Indihar, Maria Veronica, Brown, Cynthia D., Anstead, Michael, Bilodeau, Lara, Sicilian, Leonard, Jain, Manu, Tolle, James Jerome, Moffett, Kathryn, Nasr, Samya, Taylor-Cousar, Jennifer, Barto, Tara Lynn, Antos, Nicholas, Rogers, John S., Quick, Bryon, Thompson, Henry R., Sawicki, Gregory, Barnett, Bruce, Zanni, Robert L., Smith, Thomas C., Schultz, Karen D., Keating, Claire, Flume, Patrick, Omlor, Gregory J., Ashare, Alix, Voter, Karen, Mehdi, Nighat, Ortiz, Maria Gabriela Tupayachi, Gardner, Tonia E., Boas, Steven R., Messore, Barbara, Zemanick, Edith, Jain, Raksha, McCarthy, Michael, Kissner, Dana G., Patel, Kapilkumar, McNamara, John, Philley, Julie, Berlinski, Ariel, Calimano, Francisco J., Chin, Terry, Conrad, Douglas, Daines, Cori, Raissy, Hengameh H., Keens, Thomas G., Lascano, Jorge E., McWilliams, Bennie, Morrissey, Brian, Reyes, Santiago, Chittivelu, Subramanyam, Hussain, Sabiha, Stone, Arvey, Wallace, James, Klingsberg, Ross, Biller, Julie A., Bui, Stephanie, Sommerburg, Olaf, Bignamini, Elisabetta, Collura, Mirella, Biner, Reta Fischer, Moller, Alexander, Salvatore, Donatello, Belleguic, Chantal, Bentur, Lea, Efrati, Ori, Kerem, Eitan, Prais, Dario, Gallego, Esther Quintana, Barry, Peter, Livnat-Levanon, Galit, Asensi, Jose Ramon Villa, Armstrong, David Stuart, de la Cruz, Oscar Asensio, Gilchrist, Francis, Tullis, Diana Elizabeth, Quon, Bradley, Lands, Larry C., Morrison, Nancy, Lavoie, Annick, Linnane, Barry, Elidemir, Okan, Ringshausen, Felix, Kappler, Matthias, Hebestreit, Helge, Mainz, Jochen, Kiefer, Alexander, Koerner-Rettberg, Cordula, Staab, Doris, Gleiber, Wolfgang, Pressler, Tacjana, Stehling, Florian, Hector, Andreas, Sutharsan, Sivagurunathan, Naehrlich, Lutz, Fischer, Rainald, Downey, Damian, Davies, Jane Carolyn, Ketchell, Robert Ian, Carroll, Mary Patricia, Doe, Simon, MacGregor, Gordon, Nash, Edward Fairbairn, Withers, Nicholas, Peckham, Daniel Gavin, Ledson, Martin James, Kansra, Sonal, Lee, Timothy William Rayner, Delaisi, Bertrand, Rault, Gilles, Le Bihan, Jean, Hubert, Dominique, Fajac, Isabelle, Sermet-Gaudelus, Isabelle, Bakker, Marleen, Arets, Bert, De Boeck, Christiane, Chiron, Raphael, Reix, Philippe, Mainguy, Catherine, van Braeckel, Eva, Malfroot, Anne, Durieu, Isabelle, Dufeu, Nadine Desmazes, Prevotat, Anne, van der Meer, Renske, Merkus, Petrus, Weersink, E.J.M., Gomez-Aguero, Isabel Barrio, Gartner, Silvia, Jover, Amparo Sole, Fernandez, Antonio Alvarez, Cox, Desmond William, McKone, Edward F., Plant, Barry James, Selvadurai, Hiranjan, Bowler, Simon David, Wainwright, Claire Elizabeth, Smith, Daniel, Middleton, Peter Gordon, Wilson, John William, Volpi, Sonia, Colombo, Carla, Fabrizzi, Benedetta, Lucidi, Vincenzina, Cresta, Federico, Cucchiara, Salvatore, Eber, Ernst, Ellemunter, Helmut, Huttegger, Isidor, Hjelte, Lena, Krantz, Christina, Gilljam, Marita, Flume, Patrick A, Downey, Damian G, Brown, Cynthia, De Boeck, Kris, Sawicki, Gregory S, Chang, Philip, Paz-Diaz, Hildegarde, Rubin, Jaime L, Yang, Yoojung, Hu, Xingdi, Pasta, David J, Millar, Stefanie J, Campbell, Daniel, Wang, Xin, Ahluwalia, Neil, Owen, Caroline A, and Wainwright, Claire E
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- 2021
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3. Chronic Pulmonary Silicone Embolism Syndrome Following Saline Breast Implants
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Smith, Jorden E., primary, Amariei, Dianna E., additional, Herazo-Maya, Jose, additional, Niyazi, Sara, additional, Ruffolo, Robert F., additional, Patel, Kapilkumar N., additional, Quereshi, Muhammad R., additional, Abraham, Jerrold, additional, and Bandyopadhyay, Debabrata, additional
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- 2023
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4. 1513: CENTRAL CANNULATION OF VA ECMO FOR SEVERE PULMONARY HYPERTENSION AND CARDIOGENIC SHOCK
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Shankar, Shruti, Restrepo, Ricardo, Bandyopadhyay, Debabrata, Sharma, Nirmal, and Patel, Kapilkumar
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- 2022
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5. A 50-gene high-risk profile predictive of COVID-19 and Idiopathic Pulmonary Fibrosis mortality originates from a molecular imbalance in monocyte and T-cell subsets that reverses in survivors with post-COVID-19 Interstitial Lung Disease
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Tourki, Bochra, primary, Jia, Minxue, additional, Karampitsakos, Theodoros, additional, Vera, Iset M, additional, Arsenault, Alyssa, additional, Marlin, Krystin, additional, Perrot, Carole Y, additional, Allen, Dylan, additional, Farsaei, Forouzandeh, additional, Rutenberg, David, additional, Bandyopadhyay, Debabrata, additional, Restrepo, Ricardo, additional, Qureshi, Muhammad R., additional, Patel, Kapilkumar, additional, Tzouvelekis, Argyrios, additional, Kapetanaki, Maria, additional, Juan-Guardela, Brenda, additional, Kim, Kami, additional, Benos, Panayiotis V, additional, and Herazo-Maya, Jose, additional
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- 2023
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6. Mast-Cell Expressed Membrane Protein-1 (MCEMP1) is expressed in classical monocytes and alveolar macrophages in Idiopathic Pulmonary Fibrosis and regulates cell chemotaxis, adhesion, and migration in a TGFβ dependent manner.
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Perrot, Carole Y, primary, Karampitsakos, Theodoros, additional, Unterman, Avraham, additional, Adams, Taylor, additional, Marlin, Krystin, additional, Arsenault, Alyssa, additional, Zhao, Amy, additional, Kaminski, Naftali, additional, Katlaps, Gundars, additional, Patel, Kapilkumar, additional, Bandyopadhyay, Debabrata, additional, and Herazo-Maya, Jose D, additional
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- 2023
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7. Aberrant Expression of ACO1 in Vasculatures Parallels Progression of Idiopathic Pulmonary Fibrosis
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Fukumoto, Jutaro, primary, Lin, Muling, additional, Banday, Mudassir Meraj, additional, Patil, Sahebgowda Sidramagowda, additional, Krishnamurthy, Sudarshan, additional, Breitzig, Mason, additional, Soundararajan, Ramani, additional, Galam, Lakshmi, additional, Narala, Venkata Ramireddy, additional, Johns, Colleen, additional, Patel, Kapilkumar, additional, Dunning, John, additional, Lockey, Richard F., additional, Sharma, Nirmal S., additional, and Kolliputi, Narasaiah, additional
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- 2022
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8. Successful Treatment of a Patient With Severe COVID-19 Using an Integrated Approach Addressing Mast Cells and Their Mediators
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Theoharides, Theoharis C., primary, Guerra, Lucy, additional, and Patel, Kapilkumar, additional
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- 2022
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9. 1513: CENTRAL CANNULATION OF VA ECMO FOR SEVERE PULMONARY HYPERTENSION AND CARDIOGENIC SHOCK
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Shankar, Shruti, primary, Restrepo, Ricardo, additional, Bandyopadhyay, Debabrata, additional, Sharma, Nirmal, additional, and Patel, Kapilkumar, additional
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- 2021
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10. Addressing Inherited Predisposition for Breast Cancer in Transplant Recipients
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YANG, RACHEL L., KURIAN, ALLISON W., WINTON, LISA M., WEILL, DAVID, PATEL, KAPILKUMAR, KINGHAM, KERRY, and WAPNIR, IRENE L.
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- 2016
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11. Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study
- Author
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Flume, Patrick A, primary, Biner, Reta Fischer, additional, Downey, Damian G, additional, Brown, Cynthia, additional, Jain, Manu, additional, Fischer, Rainald, additional, De Boeck, Kris, additional, Sawicki, Gregory S, additional, Chang, Philip, additional, Paz-Diaz, Hildegarde, additional, Rubin, Jaime L, additional, Yang, Yoojung, additional, Hu, Xingdi, additional, Pasta, David J, additional, Millar, Stefanie J, additional, Campbell, Daniel, additional, Wang, Xin, additional, Ahluwalia, Neil, additional, Owen, Caroline A, additional, Wainwright, Claire E, additional, Gibson, Ronald L., additional, Rowe, Steven M., additional, Lechtzin, Noah, additional, Ahrens, Richard C., additional, McCoy, Karen S., additional, Aitken, Moira, additional, Donaldson, Scott H., additional, McBennett, Kimberly Ann, additional, Pilewski, Joseph M., additional, Billings, Joanne, additional, Milla, Carlos, additional, Rubenstein, Ronald, additional, Rosenbluth, Daniel Brian, additional, Linnemann, Rachel, additional, Powers, Michael R., additional, Fortner, Christopher, additional, Frederick, Carla Anne, additional, Liou, Theodore G., additional, Black, Philip, additional, Wang, Janice, additional, Colombo, John L., additional, Berdella, Maria, additional, Indihar, Maria Veronica, additional, Brown, Cynthia D., additional, Anstead, Michael, additional, Bilodeau, Lara, additional, Sicilian, Leonard, additional, Tolle, James Jerome, additional, Moffett, Kathryn, additional, Nasr, Samya, additional, Taylor-Cousar, Jennifer, additional, Barto, Tara Lynn, additional, Antos, Nicholas, additional, Rogers, John S., additional, Quick, Bryon, additional, Thompson, Henry R., additional, Sawicki, Gregory, additional, Barnett, Bruce, additional, Zanni, Robert L., additional, Smith, Thomas C., additional, Schultz, Karen D., additional, Keating, Claire, additional, Flume, Patrick, additional, Omlor, Gregory J., additional, Ashare, Alix, additional, Voter, Karen, additional, Mehdi, Nighat, additional, Ortiz, Maria Gabriela Tupayachi, additional, Gardner, Tonia E., additional, Boas, Steven R., additional, Messore, Barbara, additional, Zemanick, Edith, additional, Jain, Raksha, additional, McCarthy, Michael, additional, Kissner, Dana G., additional, Patel, Kapilkumar, additional, McNamara, John, additional, Philley, Julie, additional, Berlinski, Ariel, additional, Calimano, Francisco J., additional, Chin, Terry, additional, Conrad, Douglas, additional, Daines, Cori, additional, Raissy, Hengameh H., additional, Keens, Thomas G., additional, Lascano, Jorge E., additional, McWilliams, Bennie, additional, Morrissey, Brian, additional, Reyes, Santiago, additional, Chittivelu, Subramanyam, additional, Hussain, Sabiha, additional, Stone, Arvey, additional, Wallace, James, additional, Klingsberg, Ross, additional, Biller, Julie A., additional, Bui, Stephanie, additional, Sommerburg, Olaf, additional, Bignamini, Elisabetta, additional, Collura, Mirella, additional, Moller, Alexander, additional, Salvatore, Donatello, additional, Belleguic, Chantal, additional, Bentur, Lea, additional, Efrati, Ori, additional, Kerem, Eitan, additional, Prais, Dario, additional, Gallego, Esther Quintana, additional, Barry, Peter, additional, Livnat-Levanon, Galit, additional, Asensi, Jose Ramon Villa, additional, Armstrong, David Stuart, additional, de la Cruz, Oscar Asensio, additional, Gilchrist, Francis, additional, Tullis, Diana Elizabeth, additional, Quon, Bradley, additional, Lands, Larry C., additional, Morrison, Nancy, additional, Lavoie, Annick, additional, Linnane, Barry, additional, Elidemir, Okan, additional, Ringshausen, Felix, additional, Kappler, Matthias, additional, Hebestreit, Helge, additional, Mainz, Jochen, additional, Kiefer, Alexander, additional, Koerner-Rettberg, Cordula, additional, Staab, Doris, additional, Gleiber, Wolfgang, additional, Pressler, Tacjana, additional, Stehling, Florian, additional, Hector, Andreas, additional, Sutharsan, Sivagurunathan, additional, Naehrlich, Lutz, additional, Downey, Damian, additional, Davies, Jane Carolyn, additional, Ketchell, Robert Ian, additional, Carroll, Mary Patricia, additional, Doe, Simon, additional, MacGregor, Gordon, additional, Nash, Edward Fairbairn, additional, Withers, Nicholas, additional, Peckham, Daniel Gavin, additional, Ledson, Martin James, additional, Kansra, Sonal, additional, Lee, Timothy William Rayner, additional, Delaisi, Bertrand, additional, Rault, Gilles, additional, Le Bihan, Jean, additional, Hubert, Dominique, additional, Fajac, Isabelle, additional, Sermet-Gaudelus, Isabelle, additional, Bakker, Marleen, additional, Arets, Bert, additional, De Boeck, Christiane, additional, Chiron, Raphael, additional, Reix, Philippe, additional, Mainguy, Catherine, additional, van Braeckel, Eva, additional, Malfroot, Anne, additional, Durieu, Isabelle, additional, Dufeu, Nadine Desmazes, additional, Prevotat, Anne, additional, van der Meer, Renske, additional, Merkus, Petrus, additional, Weersink, E.J.M., additional, Gomez-Aguero, Isabel Barrio, additional, Gartner, Silvia, additional, Jover, Amparo Sole, additional, Fernandez, Antonio Alvarez, additional, Cox, Desmond William, additional, McKone, Edward F., additional, Plant, Barry James, additional, Selvadurai, Hiranjan, additional, Bowler, Simon David, additional, Wainwright, Claire Elizabeth, additional, Smith, Daniel, additional, Middleton, Peter Gordon, additional, Wilson, John William, additional, Volpi, Sonia, additional, Colombo, Carla, additional, Fabrizzi, Benedetta, additional, Lucidi, Vincenzina, additional, Cresta, Federico, additional, Cucchiara, Salvatore, additional, Eber, Ernst, additional, Ellemunter, Helmut, additional, Huttegger, Isidor, additional, Hjelte, Lena, additional, Krantz, Christina, additional, and Gilljam, Marita, additional
- Published
- 2021
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12. Acute Respiratory Distress Syndrome
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Patel, Kapilkumar, primary and Parsons, Polly E., additional
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- 2007
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13. Contributors
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Alam, Hasan B., primary, Allen, Gil, additional, Bekes, Carolyn E., additional, Berry, Jonathan, additional, Bickler, Philip E., additional, Blum, Joshua, additional, Bonar, James P., additional, Bonovich, David C., additional, Bowyer, Mark W., additional, Burkhardt, Dan, additional, Burris, David G., additional, Campbell, Lundy J., additional, Canham, E. Michael, additional, Cantrill, Stephen V., additional, Chen, Lee-lynn, additional, Chertow, Glenn M., additional, Cook, Deborah R., additional, Crommett, John W., additional, Crookes, Bruce, additional, Danai, Pajman A., additional, Deutschman, Clifford S., additional, Dixon, Anne, additional, Donnelly, Thomas J., additional, Dotson, Rachel H., additional, Fernandez, Enrique, additional, Fogarty, Patrick F., additional, Ganter, Michael T., additional, Garcia, Joel A., additional, Girod, Carlos E., additional, Gordon, Noah E., additional, Gropper, Michael A., additional, Gutsche, Jacob T., additional, Habeeb, Katherine, additional, Haenel, James, additional, Haerle, Derek, additional, Haight, Matthew J., additional, Hanley, Michael E., additional, Hanson, C. William, additional, Harzstark, Andrea, additional, Hassell, Kathryn L., additional, Heffner, John E., additional, Jacobs, Richard, additional, Jacobson, James L., additional, Jeng, James C., additional, Kaminsky, David A., additional, Kasperbauer, Shannon, additional, Kaw, Dinkar, additional, Kohl, Benjamin A., additional, Kozar, Rosemary A., additional, Kulig, Ken, additional, Lane, Rondall, additional, Lapinsky, Stephen E., additional, Lee, William Eng, additional, Linas, Stuart L., additional, Liu, Kathleen D., additional, Liu, Linda L., additional, Marcy, Theodore W., additional, Marden, Brian T., additional, Markovchick, Vincent J., additional, Marx, John A., additional, McDermott, Michael T., additional, Mehler, Philip S., additional, Messenger, John, additional, Minken, Stanley L., additional, Misset, Benoit, additional, Moore, Frederick A., additional, Morris, Amy E., additional, Moss, Marc, additional, Ng, Victor, additional, Niemann, Claus U., additional, Pao, Alan C., additional, Pardo, Manuel, additional, Parsons, Polly E., additional, Patel, Kapilkumar, additional, Perlstein, Jon, additional, Pichakron, Kullada O., additional, Peirson, Ryan P., additional, Pittet, Jean-François, additional, Polish, Louis B., additional, Pons, Peter T., additional, Rebuck, Jill A., additional, Reves, Randall, additional, Ritter, E. Matt, additional, Rozwadowski, Jeanne M., additional, Sava, Jack, additional, Savel, Richard H., additional, Schnapp, Lynn M., additional, Schulman, Peter M., additional, Shapiro, Joseph I., additional, Shimabukuro, David, additional, Sidlow, Stuart F., additional, Spevetz, Antoinette, additional, Stralovich-Romani, Annette, additional, Taylor, John M., additional, Thomas, Marshall, additional, Varma, Madhulika G., additional, Velayos, Fernando, additional, Wang, Jennifer Y., additional, Welsh, Carolyn H., additional, Wiedeman, James E., additional, Wiener-Kronish, Jeanine P., additional, Williams, Danny C., additional, Yang, Jon, additional, Young, Michael, additional, and Zamora, Martin R., additional
- Published
- 2007
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14. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial
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Heijerman, Harry G M, primary, McKone, Edward F, additional, Downey, Damian G, additional, Van Braeckel, Eva, additional, Rowe, Steven M, additional, Tullis, Elizabeth, additional, Mall, Marcus A, additional, Welter, John J, additional, Ramsey, Bonnie W, additional, McKee, Charlotte M, additional, Marigowda, Gautham, additional, Moskowitz, Samuel M, additional, Waltz, David, additional, Sosnay, Patrick R, additional, Simard, Christopher, additional, Ahluwalia, Neil, additional, Xuan, Fengjuan, additional, Zhang, Yaohua, additional, Taylor-Cousar, Jennifer L, additional, McCoy, Karen S, additional, McCoy, Karen, additional, Donaldson, Scott, additional, Walker, Seth, additional, Chmiel, James, additional, Rubenstein, Ronald, additional, Froh, Deborah K., additional, Neuringer, Isabel, additional, Jain, Manu, additional, Moffett, Kathryn, additional, Taylor-Cousar, Jennifer L., additional, Barnett, Bruce, additional, Mueller, Gary, additional, Flume, Patrick, additional, Livingston, Floyd, additional, Mehdi, Nighat, additional, Teneback, Charlotte, additional, Welter, John, additional, Jain, Raksha, additional, Kissner, Dana, additional, Patel, Kapilkumar, additional, Calimano, Francisco J., additional, Johannes, Jimmy, additional, Daines, Cori, additional, Keens, Thomas, additional, Scher, Herschel, additional, Chittivelu, Subramanyam, additional, Reddivalam, Sudhakar, additional, Klingsberg, Ross Carl, additional, Johnson, Larry G., additional, Verhulst, Stijn, additional, Macedo, Patricia, additional, Downey, Damien, additional, Connett, Gary, additional, Nash, Edward, additional, Withers, Nicholas, additional, Lee, Timothy, additional, Bakker, Marleen, additional, Heijerman, Harry, additional, Vermeulen, Francois, additional, Knoop, Christiane, additional, De Wachter, Elke, additional, van der Meer, Renske, additional, Merkus, Petrus, additional, and Majoor, Christof, additional
- Published
- 2019
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15. Characterization of cell proliferation in idiopathic pulmonary fibrosis
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Fukumoto, Jutaro, primary, Patil, Sahebgowda, additional, Krishnamurthy, Sudarshan, additional, Galam, Lakshmi, additional, Sharma, Nirmal, additional, Patel, Kapilkumar, additional, Hooker, Robert, additional, Dunning, John, additional, Lockey, Richard F., additional, and Kolliputi, Narasaiah, additional
- Published
- 2019
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16. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508delmutation: a double-blind, randomised, phase 3 trial
- Author
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Heijerman, Harry G M, McKone, Edward F, Downey, Damian G, Van Braeckel, Eva, Rowe, Steven M, Tullis, Elizabeth, Mall, Marcus A, Welter, John J, Ramsey, Bonnie W, McKee, Charlotte M, Marigowda, Gautham, Moskowitz, Samuel M, Waltz, David, Sosnay, Patrick R, Simard, Christopher, Ahluwalia, Neil, Xuan, Fengjuan, Zhang, Yaohua, Taylor-Cousar, Jennifer L, McCoy, Karen S, McCoy, Karen, Donaldson, Scott, Walker, Seth, Chmiel, James, Rubenstein, Ronald, Froh, Deborah K., Neuringer, Isabel, Jain, Manu, Moffett, Kathryn, Taylor-Cousar, Jennifer L., Barnett, Bruce, Mueller, Gary, Flume, Patrick, Livingston, Floyd, Mehdi, Nighat, Teneback, Charlotte, Welter, John, Jain, Raksha, Kissner, Dana, Patel, Kapilkumar, Calimano, Francisco J., Johannes, Jimmy, Daines, Cori, Keens, Thomas, Scher, Herschel, Chittivelu, Subramanyam, Reddivalam, Sudhakar, Klingsberg, Ross Carl, Johnson, Larry G., Verhulst, Stijn, Macedo, Patricia, Downey, Damien, Connett, Gary, Nash, Edward, Withers, Nicholas, Lee, Timothy, Bakker, Marleen, Heijerman, Harry, Vermeulen, Francois, Van Braeckel, Eva, Knoop, Christiane, De Wachter, Elke, van der Meer, Renske, Merkus, Petrus, and Majoor, Christof
- Abstract
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the basic defect caused by CFTR mutations. Improvements in health outcomes have been achieved with the combination of a CFTR corrector and potentiator in people with cystic fibrosis homozygous for the F508delmutation. The addition of elexacaftor (VX-445), a next-generation CFTR corrector, to tezacaftor plus ivacaftor further improved F508del-CFTR function and clinical outcomes in a phase 2 study in people with cystic fibrosis homozygous for the F508delmutation.
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- 2019
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17. Carmustine-Associated Pneumonitis Presenting as a Unilateral Pleural Effusion
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Lin, Chunrong, primary, Patel, Kapilkumar, additional, and Hsu, Joe, additional
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- 2014
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18. Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR(EXTEND): an open-label extension study
- Author
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Flume, Patrick A, Biner, Reta Fischer, Downey, Damian G, Brown, Cynthia, Jain, Manu, Fischer, Rainald, De Boeck, Kris, Sawicki, Gregory S, Chang, Philip, Paz-Diaz, Hildegarde, Rubin, Jaime L, Yang, Yoojung, Hu, Xingdi, Pasta, David J, Millar, Stefanie J, Campbell, Daniel, Wang, Xin, Ahluwalia, Neil, Owen, Caroline A, Wainwright, Claire E, Gibson, Ronald L., Rowe, Steven M., Lechtzin, Noah, Ahrens, Richard C., McCoy, Karen S., Aitken, Moira, Donaldson, Scott H., McBennett, Kimberly Ann, Pilewski, Joseph M., Billings, Joanne, Milla, Carlos, Rubenstein, Ronald, Rosenbluth, Daniel Brian, Linnemann, Rachel, Powers, Michael R., Fortner, Christopher, Frederick, Carla Anne, Liou, Theodore G., Black, Philip, Wang, Janice, Colombo, John L., Berdella, Maria, Indihar, Maria Veronica, Brown, Cynthia D., Anstead, Michael, Bilodeau, Lara, Sicilian, Leonard, Jain, Manu, Tolle, James Jerome, Moffett, Kathryn, Nasr, Samya, Taylor-Cousar, Jennifer, Barto, Tara Lynn, Antos, Nicholas, Rogers, John S., Quick, Bryon, Thompson, Henry R., Sawicki, Gregory, Barnett, Bruce, Zanni, Robert L., Smith, Thomas C., Schultz, Karen D., Keating, Claire, Flume, Patrick, Omlor, Gregory J., Ashare, Alix, Voter, Karen, Mehdi, Nighat, Ortiz, Maria Gabriela Tupayachi, Gardner, Tonia E., Boas, Steven R., Messore, Barbara, Zemanick, Edith, Jain, Raksha, McCarthy, Michael, Kissner, Dana G., Patel, Kapilkumar, McNamara, John, Philley, Julie, Berlinski, Ariel, Calimano, Francisco J., Chin, Terry, Conrad, Douglas, Daines, Cori, Raissy, Hengameh H., Keens, Thomas G., Lascano, Jorge E., McWilliams, Bennie, Morrissey, Brian, Reyes, Santiago, Chittivelu, Subramanyam, Hussain, Sabiha, Stone, Arvey, Wallace, James, Klingsberg, Ross, Biller, Julie A., Bui, Stephanie, Sommerburg, Olaf, Bignamini, Elisabetta, Collura, Mirella, Biner, Reta Fischer, Moller, Alexander, Salvatore, Donatello, Belleguic, Chantal, Bentur, Lea, Efrati, Ori, Kerem, Eitan, Prais, Dario, Gallego, Esther Quintana, Barry, Peter, Livnat-Levanon, Galit, Asensi, Jose Ramon Villa, Armstrong, David Stuart, de la Cruz, Oscar Asensio, Gilchrist, Francis, Tullis, Diana Elizabeth, Quon, Bradley, Lands, Larry C., Morrison, Nancy, Lavoie, Annick, Linnane, Barry, Elidemir, Okan, Ringshausen, Felix, Kappler, Matthias, Hebestreit, Helge, Mainz, Jochen, Kiefer, Alexander, Koerner-Rettberg, Cordula, Staab, Doris, Gleiber, Wolfgang, Pressler, Tacjana, Stehling, Florian, Hector, Andreas, Sutharsan, Sivagurunathan, Naehrlich, Lutz, Fischer, Rainald, Downey, Damian, Davies, Jane Carolyn, Ketchell, Robert Ian, Carroll, Mary Patricia, Doe, Simon, MacGregor, Gordon, Nash, Edward Fairbairn, Withers, Nicholas, Peckham, Daniel Gavin, Ledson, Martin James, Kansra, Sonal, Lee, Timothy William Rayner, Delaisi, Bertrand, Rault, Gilles, Le Bihan, Jean, Hubert, Dominique, Fajac, Isabelle, Sermet-Gaudelus, Isabelle, Bakker, Marleen, Arets, Bert, De Boeck, Christiane, Chiron, Raphael, Reix, Philippe, Mainguy, Catherine, van Braeckel, Eva, Malfroot, Anne, Durieu, Isabelle, Dufeu, Nadine Desmazes, Prevotat, Anne, van der Meer, Renske, Merkus, Petrus, Weersink, E.J.M., Gomez-Aguero, Isabel Barrio, Gartner, Silvia, Jover, Amparo Sole, Fernandez, Antonio Alvarez, Cox, Desmond William, McKone, Edward F., Plant, Barry James, Selvadurai, Hiranjan, Bowler, Simon David, Wainwright, Claire Elizabeth, Smith, Daniel, Middleton, Peter Gordon, Wilson, John William, Volpi, Sonia, Colombo, Carla, Fabrizzi, Benedetta, Lucidi, Vincenzina, Cresta, Federico, Cucchiara, Salvatore, Eber, Ernst, Ellemunter, Helmut, Huttegger, Isidor, Hjelte, Lena, Krantz, Christina, and Gilljam, Marita
- Abstract
Tezacaftor–ivacaftor is an approved cystic fibrosis transmembrane conductance regulator (CFTR) modulator shown to be efficacious and generally safe and well tolerated over 8–24 weeks in phase 3 clinical studies in participants aged 12 years or older with cystic fibrosis homozygous for the Phe508del CFTRmutation (F/F; study 661-106 [EVOLVE]) or heterozygous for the Phe508del CFTRmutation and a residual function mutation (F/RF; study 661-108 [EXPAND]). Longer-term (>24 weeks) safety and efficacy of tezacaftor–ivacaftor has not been assessed in clinical studies. Here, we present results of study 661-110 (EXTEND), a 96-week open-label extension study that assessed long-term safety, tolerability, and efficacy of tezacaftor–ivacaftor in participants aged 12 years or older with cystic fibrosis who were homozygous or heterozygous for the Phe508del CFTRmutation.
- Published
- 2021
- Full Text
- View/download PDF
19. Mast-cell expressed membrane protein-1 is expressed in classical monocytes and alveolar macrophages in idiopathic pulmonary fibrosis and regulates cell chemotaxis, adhesion, and migration in a TGFβ-dependent manner.
- Author
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Perrot CY, Karampitsakos T, Unterman A, Adams T, Marlin K, Arsenault A, Zhao A, Kaminski N, Katlaps G, Patel K, Bandyopadhyay D, and Herazo-Maya JD
- Subjects
- Humans, Monocytes metabolism, Membrane Proteins metabolism, Chemotaxis, Mast Cells metabolism, Transforming Growth Factor beta metabolism, Macrophages, Alveolar metabolism, Idiopathic Pulmonary Fibrosis genetics, Idiopathic Pulmonary Fibrosis metabolism
- Abstract
Mast-cell expressed membrane protein-1 (MCEMP1) is higher in patients with idiopathic pulmonary fibrosis (IPF) with an increased risk of death. Here we aimed to establish the mechanistic role of MCEMP1 in pulmonary fibrosis. We identified increased MCEMP1 expression in classical monocytes and alveolar macrophages in IPF compared with controls. MCEMP1 is upregulated by transforming growth factor beta (TGFβ) at the mRNA and protein levels in monocytic leukemia THP-1 cells. TGFβ-mediated MCEMP1 upregulation results from the cooperation of SMAD3 and SP1 via concomitant binding to SMAD3/SP1 cis- regulatory elements within the MCEMP1 promoter. We also found that MCEMP1 regulates TGFβ-mediated monocyte chemotaxis, adhesion, and migration. Our results suggest that MCEMP1 may regulate the migration and transition of monocytes to monocyte-derived alveolar macrophages during pulmonary fibrosis development and progression. NEW & NOTEWORTHY MCEMP1 is highly expressed in circulating classical monocytes and alveolar macrophages in IPF, is regulated by TGFβ, and participates in the chemotaxis, adhesion, and migration of circulating monocytes by modulating the effect of TGFβ in RHO activity.
- Published
- 2024
- Full Text
- View/download PDF
20. Mast-Cell Expressed Membrane Protein-1 (MCEMP1) is expressed in classical monocytes and alveolar macrophages in Idiopathic Pulmonary Fibrosis and regulates cell chemotaxis, adhesion, and migration in a TGFβ dependent manner.
- Author
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Perrot CY, Karampitsakos T, Unterman A, Adams T, Marlin K, Arsenault A, Zhao A, Kaminski N, Katlaps G, Patel K, Bandyopadhyay D, and Herazo-Maya JD
- Abstract
Background: Mast-Cell Expressed Membrane Protein-1 (MCEMP1) is higher in Idiopathic Pulmonary Fibrosis (IPF) patients with increased risk of death and poor outcomes. Here we seek to establish the mechanistic role of MCEMP1 in pulmonary fibrosis., Methods: MCEMP1 expression was analyzed by single-cell RNA sequencing, immunofluorescence in Peripheral Blood Mononuclear Cells (PBMC) as well as in lung tissues from IPF patients and controls. Chromatin Immunoprecipitation (ChiP) and Proximity Ligation Assay (PLA) were used to study the transcriptional regulation of MCEMP1 . Transient RNA interference and lentivirus transduction were used to knockdown and knock-in MCEMP1 in THP-1 cells to study chemotaxis, adhesion, and migration. Bulk RNA sequencing was used to identify the mechanisms by which MCEMP1 participates in monocyte function. Active RHO pull-down assay was used to validate bulk RNA sequencing results., Results: We identified increased MCEMP1 expression in classical monocytes and alveolar macrophages in IPF compared to controls. MCEMP1 was upregulated by TGFβ at the mRNA and protein levels in THP-1. TGFβ-mediated MCEMP1 upregulation results from the cooperation of SMAD3 and SP1 via concomitant binding to SMAD3/SP1 cis -regulatory elements within the MCEMP1 promoter. In terms of its function, we found that MCEMP1 regulates TGFβ-mediated monocyte chemotaxis, adhesion, and migration. 400 differentially expressed genes were found to increase after TGFβ stimulation of THP-1, further increased in MCEMP1 knock-in cells treated with TGFβ and decreased in MCEMP1 knockdown cells treated with TGFβ. GO annotation analysis of these genes showed enrichment for positive regulation of RHO GTPase activity and signal transduction. While TGFβ enhanced RHO GTPase activity in THP-1 cells, this effect was attenuated following MCEMP1 knockdown., Conclusion: MCEMP1 is highly expressed in circulating classical monocytes and alveolar macrophages in IPF. MCEMP1 is regulated by TGFβ and participates in the chemotaxis, adhesion, and migration of circulating monocytes by modulating the effect of TGFβ in RHO activity. Our results suggest that MCEMP1 may regulate the migration and transition of monocytes to monocyte-derived alveolar macrophages during pulmonary fibrosis development and progression.
- Published
- 2023
- Full Text
- View/download PDF
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