Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor–tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials

Authors: Krick, Stefanie, McCoy, Karen, Goss, Christopher, Weiner, Daniel, Billings, Joanne, Ren, Clement, Atkinson, Jeffrey, Linnemann, Rachel, Trimble, Aaron, Frederick, Carla, Liou, Theodore, Escobar, Hugo, Wang, Janice, Deschamps, Ashley, Froh, Deborah, Chmiel, James, Wurth, Mark, Yonker, Lael, Jain, Manu, Millard, Susan, Tolle, James, Filbrun, Amy, Taylor-Cousar, Jennifer, Ruiz, Fadel, Brown, Cynthia, Quick, Bryon, Brown, Perry, Kennedy, John, Ruddy, Jennifer, Shkolnik, Boris, Pancham, Krishna, DiMango, Emily, Fiel, Stanley, Mueller, Gary, Livingston, Floyd, Braun, Andrew, O'Sullivan, Brian, Dubin, Patricia, Teneback, Charlotte, Kremer, Ted, Welter, John, Hoppe, Jordana, Jain, Raksha, Calimano, Francisco, Johannes, Jimmy, Daines, Cori, Green, Deanna, Keens, Thomas, Trillo-Alvarez, Cesar, Scher, Herschel, Mermis, Joel, Morrissey, Brian, Wallace, James, Barrios, Christopher, Callison, John, Holliday, Zachary, Klingsberg, Ross, Efrati, Ori, Cohen-Cymberknoh, Malena, Prais, Dario, Azevedo, Pilar, Quintana-Gallego, Esther, Wong, Janice, Mondejar-Lopez, Pedro, Lopez Neyra, Alejandro, Amorim, Adelina, Sivam, Sheila, Asensio de la Cruz, Oscar, Waller, Michael, Forseen, Caralee, Marion, Chad, Dittrich, Anna-Maria, Lysinger, Jerimiah, Casserly, Brian, Luna, Carmen, Brown, Sarah, Singer, Jonathan, Schellhase, Dennis, Roberts, Jon, King, Christopher, Byrnes, Catherine, Ringshausen, Felix, Hebestreit, Helge, Lorenz, Michael, Ruppel, Renate, Drevinek, Pavel, Dillenhofer, Stefanie, Stahl, Mirjam, Gleiber, Wolfgang, Homola, Lukas, Naehrig, Susanne, Stehling, Florian, Sutharsan, Sivagurunathan, Fischer, Rainald, Poplawska, Krystyna, Haworth, Charles, Simmonds, Nicholas, Urquhart, Don, Duckers, Jamie, Carroll, Mary, Barry, Peter, Doe, Simon, Hilliard, Tom, MacGregor, Gordon, Patel, Neil, Withers, Nicholas, Peckham, Daniel, Ledson, Martin, Koser, Usma, Gartner, Silvia, Blitz Castro, Enrique, Sole Jover, Amparo, Cox, Desmond, McKone, Edward, Plant, Barry, Williamson, Michael, Elnazir, Basil, Barbosa, Telma, Cols Roig, Maria, Burr, Lucy, Henderson, Daniel, Middleton, Peter, Kotsimbos, Tom, De Monestrol, Isabelle, Gilljam, Marita, Lindberg, Ulrika, Schwarz, Carsten, Bode, Sebastian, Epton, Michael, Halasz, Adrien, Laki, Istvan, O'Carroll, Mark, Fortner, Christopher, Bilodeau, Lara, Lapey, Allen, Moffett, Kathryn, Jia, Shijing, Uluer, Ahmet, Sears, Edmund, Keating, Claire, Omlor, Gregory, Mehdi, Nighat, Tupayachi Ortiz, Maria Gabriela, Messore, Barbara, Lambert, Allison, Mukadam, Zubin, Egan, Marie, Wilhelm, Andrew, Reyes, Santiago, Schaeffer, David, Szepfalusi, Zsolt, Epaud, Ralph, Leroy, Sylvie, Fischer Biner, Reta, Moeller, Alexander, Salvatore, Donatello, Johnson, Larry, Gohy, Sophie, Gramegna, Andrea, Olesen, Hanne, Tissot, Adrien, Marguet, Christophe, Desai, Maya, Owens, Louisa, Kherani, Tamizan, Sands, Dorota, Bakkeheim, Egil, Tullis, Elizabeth, Chilvers, Mark, Quon, Bradley, Lands, Larry, Parkins, Michael, Tremblay, Francois, Maitra, Anirban, Staples, Heather, Elidemir, Okan, Chatziagorou, Elpis, Tirakitsoontorn, Pornchai, Grenet, Dominique, Linnane, Barry, Nicholson, John Michael, Manika, Katerina, Tatopoulos, Aurelie, Pressler, Tacjana, Bagheri-Potthoff, Azadeh, Legg, Julian, Horsley, Alexander, Lee, Timothy, Houdouin, Veronique, Ramel, Sophie, Fajac, Isabelle, Sermet-Gaudelus, Isabelle, Kamphuis, Lieke, van der Ent, Kors, Vermeulen, Francois, Chiron, Raphael, Reix, Philippe, Van Braeckel, Eva, Knoop, Christiane, Vanderhelst, Eef, Durieu, Isabelle, Bui, Stephanie, Weiss, Laurence, Le Rouzic, Olivier, van der Meer, Renske, Roukema, Jolt, Altenburg, Josje, Robinson, Philip, Wainwright, Claire, Mulrennan, Siobhain, Clements, Barry, Taccetti, Giovanni, Cipolli, Marco, Dacco, Valeria, Fabrizzi, Benedetta, Castellani, Carlo, Eber, Ernst, Appelt, Dorothea, Studnicka, Michael, Sapiejka, Ewa, Mazurek, Henryk, Engan, Mette, Sauty, Alain, Mankikian, Julie, Yonker, Lael M ^b, Vermeulen, François ^c, Linnemann, Rachel W ^e, Braun, Andrew T ⁱ, Ramsey, Bonnie ^l, Mall, Marcus A ^{m, n, o}, Taylor-Cousar, Jennifer L ^p, McKone, Edward F ^q, Floreth, Tim ^s, Michelson, Peter ^s, Sosnay, Patrick R ^s, Nair, Nitin ^s, Zahigian, Rachel ^s, Martin, Hannah ^s, Ahluwalia, Neil ^s, Lam, Anna ^s

Source: In The Lancet Respiratory Medicine March 2025 13(3):256-271

Impact of COVID-19 infection on lung function and nutritional status amongst individuals with cystic fibrosis: A global cohort study

Authors: Kasmi, Irena, Drali, Ouardia, Burghart, Sabine, Lakatos-Krepcik, Andrea, Eder, Johannes, Jaksch, Peter, Kainz, Katharina, Kallinger, Margit, Leitner, Alexander, Mozdzen, Marta, Pfleger, Andreas, Renner, Sabine, Stadlinger, Martin, Thir, Christina, Nuriyev, Emil, Boboli, Hedwige, De Wachter, Elke, Dupont, Lieven, Gohy, Sophie, Hanssens, Laurence, Knoop, Christiane, Lammertyn, Elise, Nowé, Vicky, Pirson, Jessica, Thimmesch, Matthieu, Van Braeckel, Eva, Van Hoorenbeeck, Kim, Vanderhelst, Eef, Filho, Eduardo Piacentini, Athanazio, Rodrigo Abensur, Martins, Valéria de Carvalho, Duarte, Marta Cristina, Monte, Luciana de Freitas Velloso, de Fuccio, Marcelo Bicalho, Knabben, Adriana de Siqueira Carvalho, Melloti, Roberta, Meneses, Daniela Gois, Petrova, Guergana, Tješić-Drinković, Duška, Dugac, Andrea Vukić, Bambir, Ivan, Yiallouros, Panayiotis, Bilkova, Alena, Drevinek, Pavel, Macek, Milan, Jr, Olesen, Hanne Vebert, Pressler, Tania, Fouda, Eman Mahmoud, Nasr, Samya, Weldetsadik, Abate Yeshidinber, Al-iede, Montaha, Abdrakhmanov, Olzhas, Corvol, Harriet, Lemonnier-Videau, Lydie, Abely, Michel, Piccini, Carole Bailly, Belleguic, Chantal, Bihouee, Tiphaine, Billon, Yves, Bui, Stéphanie, Camara, Boubou, Cheraud, Marie-Christine, Chiron, Raphael, Duet, Emmanuelle Coirier, Cosson, Laure, Dalphin, Marie-Laure, Boucher, Isabelle Danner, De Miranda, Sandra, Deneuville, Eric, Dubus, Jean-Christophe, Durieu, Isabelle, Epaud, Ralph, Gerardin, Michèle, Grenet, Dominique, Houdouin, Véronique, Huet, Frédéric, Reem, Kanaan, Kessler, Romain, Languepin, Jeanne, Laurans, Muriel, Leroy, Sylvie, Llerena, Cathie, Macey, Julie, Mankikian, Julie, Marguet, Christophe, Martin, Clémence, Mely, Laurent, Mittaine, Marie, Murris-Espin, Marlène, Perisson, Caroline, Prevotat, Anne, Ramel, Sophie, Rames, Cinthia, Reix, Philippe, Revillon, Marine, Reynaud-Gaubert, Martine, Richaud-Thiriez, Bénédicte, Rittie, Jean-Luc, Scalbert-Dujardin, Manuëla, Sermet-Gaudelus, Isabelle, Storni, Véronique, Tatopoulos, Aurélie, Thouvenin, Guillaume, Troussier, Françoise, Weiss, Laurence, Wizla, Nathalie, Behl, Eva-Susanne, Brinkmann, Folke, Claßen, Martin, Graepler-Mainka, Ute, Griese, Matthias, Grübl, Armin, Hammermann, Jutta, Hebestreit, Helge, Heinzmann, Andrea, Herz, Alexander, Kiefer, Alexander, Kinder, Birte, Köster, Holger, Kuhnert, Stefan, Mainz, Jochen, Mayer, Angelika, Naehrig, Susanne, Niehues, Tim, Nüßlein, Thomas, Poplawska, Krystyna, Ringshausen, Felix, Rose, Markus, Rosenecker, Josef, Ruppel, Renate, Scharschinger, Anette, Schropp, Christian, Schwarz, Carsten, Smaczny, Christina, Sommerburg, Olaf, Sutharsan, Sivagurunathan, Stolz, Simone, Thomas, Wolfgang, Wege, Sabine, Welzenbach, Britta, Wollschläger, Bettina, Diamantea, Filia, Hatziagorou, Elpis, Manika, Katerina, Cox, Des, Elnazir, Basil, Fletcher, Godfrey, Gunaratnam, Cedric, McKone, Edward F., Plant, Barry J., Cohen-Cymberknoh, Malena, Gur, Michal, Livnat, Galit, Mei-Zahav, Meir, Amato, Annalisa, Ferrari, Gianluca, Badolato, Raffaele, Poli, Piercarlo, Battistini, Fiorella, Donati, Valentina, Bignamini, Elisabetta, Folino, Anna, Carnovale, Vincenzo, Castellani, Carlo, Casciaro, Rosaria, Cimino, Giuseppe, Cipolli, Marco, Lucca, Francesca, Collura, Mirella, Ficili, Francesca, Daccò, Valeria, Gagliano, Vanessa, Pizzamiglio, Giovanna, Mencarini, Valeria, Palladino, Nicola, Leonardi, Salvatore, Rotolo, Novella, Lucanto, Maria Cristina, Quattromano, Ester, Lucidi, Vincenzina, Majo, Fabio, Alghisi, Federico, Ciciriello, Fabiana, Manca, Antonio, Leonetti, Giuseppina, Maschio, Massimo, Messore, Barbara, Pantano, Stefano, Pisi, Giovanna, Spaggiari, Cinzia, Raia, Valeria, Laezza, Caterina, Ros, Mirco, Salvatore, Donatello, Taccetti, Giovanni, Francalanci, Michela, Vitullo, Pamela, Zolin, Anna, Aleksejeva, Elina, Malakauskas, Kestutis, Misevičiene, Valdone, Charatsi, Anna-Maria, la Barrière, Hélène De, Altenburg, Josje, Bannier, Michiel, Heijerman, Harry, Janssens, Hettie, Koppelman, Gerard, van der Meer, Renske, Merkus, Peter, Nuijsink, Marianne, Terheggen, Suzanne, van der Vaart, Hester, Wesseling, Geert-Jan, de Winter, Karin, Danevska, Ivana Arnaudova, Maretti, Tatjana Jakovska, Fustik, Stojka, Dziecichowicz-Latała, Daria, Wojsyk-Banaszak, Irena, Wozniacki, Lukasz, Amorim, Adelina, Santos, Ana Sofia Araújo, Castanhinha, Susana, Gamboa, Fernanda, Silva, Teresa Reis, Gonçalves, Fabienne, Pereira, Luísa, Ciuca, Ioana, Silva, Sónia, Csilla-Enikö, Szabo, Stan, Iustina, Amelina, Elena, Boitсova, Evgeniya, Chernyavskaya, Anastasia, Gorinova, Yuliya, Krasovskiy, Stanislav, Mukhina, Maria, Sherman, Victoria, Simonova, Olga, Kondratyeva, Elena, Bérešová, Eva, Bližnáková, Nina, Kayserová, Hana, Salobir, Barbara, Šelb, Julij, Krivec, Uroš, Fernandez, Antonio José Aguilar, Fernàndez, Antonio Alvarez, García, Félix Baranda, Aparicio, Marina Blanco, Corullón, Silvia Castillo, Cortell-Aznar, Isidoro, Pérez, Inés, Colomer, Jordi Costa i, Roig, María Cols, Pecellín, Isabel Delgado, Cáceres, Layla Diab, Paredes, Carmen Luna, Gartner, Silvia, Martínez, José Ramón Gutiérrez, Labarga, Inés Herrero, Girón-Moreno, Rosa Maria, Nogueira, Esperanza Jiménez, Ferreiro, Adelaida Lamas, Neyra, Alejandro López, Castro, Enrique Blitz, Galarraga, Laura Moreno, de Vincente, Carlos Martin, Navarro, Silvia Merlos, Nieto-Royo, Rosa, Fuster, Casilda Olveira, Pastor, Maria Dolores, Pérez-Ruiz, Estela, Prados-Sánchez, Concepción, Cancelo, Isabel Ramos, de Valbuena, Marta Ruiz, Asensi, José R. Villa, Santiago, Veronica Sanz, García, Patricia Fernández, Tawfeeq, Reem Mustafa, Banki, Adrienn, Gilljam, Marita, Krantz, Christina, Lindberg, Ulrika, Lindblad, Anders, Clarenbach, Christian, Steinack, Carolin, Hage, René, Schuurmans, Macé, Fischer, Reta, Kusche, Rachel, Rochat, Isabelle, Walter, Anna-Lena, Kamalaporn, Harutai, Hamouda, Samia, Tural, Dilber Ademhan, Ozcelik, Ugur, Asfuroğlu, Pelin, Eyüboğlu, Tuğba Şişmanlar, Aslan, Ayse Tana, Bingöl, Ayşen, Çobanoğlu, Nazan, Ozcan, Gizem, Dogru, Deniz, Gökdemir, Yasemin, KÖSE, Mehmet, Pekcan, Sevgi, Cosgriff, Rebecca, Semenchuk, Julie, Naito, Yumi, Charman, Susan C., Carr, Siobhán B, Cheng, Stephanie Y., Marshall, Bruce C., Faro, Albert, Elbert, Alexander, Gutierrez, Hector H., Goss, Christopher H., Karadag, Bulent, Burgel, Pierre-Régis, Colombo, Carla, Salvatore, Marco, Padoan, Rita, Daneau, Géraldine, Harutyunyan, Satenik, Kashirskaya, Nataliya, Kirwan, Laura, Middleton, Peter G, Ruseckaite, Rasa, de Monestrol, Isabelle, Naehrlich, Lutz, Mondejar-Lopez, Pedro, Jung, Andreas, van Rens, Jacqui, Bakkeheim, Egil, Orenti, Annalisa, Zomer-van Ommen, Domenique, da Silva-Filho, Luiz Vicente RF, Fernandes, Flavia Fonseca, Zampoli, Marco, Stephenson, Anne L.

Source: In Journal of Cystic Fibrosis September 2024 23(5):815-822

Relationship Between Lung Volumes and Heterogeneity in the Response to Elexacaftor/Tezacaftor/Ivacaftor in Patients With Cystic Fibrosis and Advanced Lung Disease

Authors: Gramegna, Andrea, Alicandro, Gianfranco, Premuda, Chiara, Lucca, Francesca, Pinali, Lucia, Retucci, Mariangela, Vespro, Valentina, Andrisani, Maria Carmela, Carraffiello, Gianpaolo, Amati, Francesco, Volpi, Sonia, Aliberti, Stefano, Cipolli, Marco, Blasi, Francesco

Source: In Chest September 2024 166(3):433-441

Efficacy of Elexacaftor–Tezacaftor–Ivacaftor on chronic rhinosinusitis in cystic fibrosis

Authors: Di Gioia, Stefano, Lucca, Francesca, Venditto, Laura, Sandri, Giulio, Tommasi, Nicola, Cipolli, Marco, Molteni, Gabriele

Source: In American Journal of Otolaryngology--Head and Neck Medicine and Surgery May-June 2024 45(3)

ESPEN-ESPGHAN-ECFS guideline on nutrition care for cystic fibrosis

Authors: Wilschanski, Michael, Munck, Anne, Carrion, Estefania, Cipolli, Marco, Collins, Sarah, Colombo, Carla, Declercq, Dimitri, Hatziagorou, Elpis, Hulst, Jessie, Kalnins, Daina, Katsagoni, Christina N., Mainz, Jochen G., Ribes-Koninckx, Carmen, Smith, Chris, Smith, Thomas, Van Biervliet, Stephanie, Chourdakis, Michael

Source: In Clinical Nutrition February 2024 43(2):413-445

The impact of elexacaftor/tezacaftor/ivacaftor therapy on the pulmonary management of adults with cystic fibrosis: An expert-based Delphi consensus

Authors: Gramegna, Andrea, Aliberti, Stefano, Calderazzo, Maria Adelaide, Casciaro, Rosaria, Ceruti, Clara, Cimino, Giuseppe, Fabrizzi, Benedetta, Lucanto, Cristina, Messore, Barbara, Pisi, Giovanna, Taccetti, Giovanni, Tarsia, Paolo, Blasi, Francesco, Cipolli, Marco

Source: In Respiratory Medicine December 2023 220

Long-term clinical outcomes of elexacaftor/tezacaftor/ivacaftor therapy in adults with cystic fibrosis and advanced pulmonary disease

Authors: Savi, Daniela, Lucca, Francesca, Tridello, Gloria, Meneghelli, Ilaria, Comello, Isabella, Tomezzoli, Sara, Signorini, Monica, Proietti, Elena, Cucchetto, Giulia, Volpi, Sonia, Cipolli, Marco

Source: In Respiratory Medicine November-December 2023 219

Traits, trends and hits of orphan drug designations in cystic fibrosis

Authors: Costa, Enrico, Girotti, Silvia, van den Ham, Hendrika A, Cipolli, Marco, van der Ent, Cornelis K, Taylor-Cousar, Jennifer L., Leufkens, Hubert G.M.

Source: In Journal of Cystic Fibrosis September 2023 22(5):949-957

Stem Cell Transplantation in Patients Affected by Shwachman-Diamond Syndrome: Expert Consensus and Recommendations From the EBMT Severe Aplastic Anaemia Working Party

Authors: Cesaro, Simone, Donadieu, Jean, Cipolli, Marco, Dalle, Jean Hugues, Styczynski, Jan, Masetti, Riccardo, Strahm, Brigitte, Mauro, Margherita, Alseraihy, Amal, Aljurf, Mahmoud, Dufour, Carlo, de la Tour, Regis Peffault

Source: In Transplantation and Cellular Therapy October 2022 28(10):637-649

Elexacaftor/Tezacaftor/Ivacaftor Efficacy in a Cohort of Italian Patients with CFTR Rare Mutations.

Authors: Lucca, Francesca, Volpi, Sonia, Ros, Mirco, Fabrizzi, Benedetta, Meneghelli, Ilaria, Bordicchia, Marica, Buniotto, Francesca, Lancini, Alessia, Brignole, Cecilia, Pauro, Francesca, Bezzerri, Valentino, Cipolli, Marco

Source: International Journal of Translational Medicine; Mar2025, Vol. 5 Issue 1, p11, 7p

Subject Terms: CYSTIC fibrosis, TREATMENT effectiveness, PULMONARY function tests

Geographic Terms: EUROPE

Long-term amikacin liposome inhalation suspension in cystic fibrosis patients with chronic P. aeruginosa infection

Authors: Bilton, Diana, Fajac, Isabelle, Pressler, Tacjana, Clancy, John Paul, Sands, Dorota, Minic, Predrag, Cipolli, Marco, Galeva, Ivanka, Solé, Amparo, Quittner, Alexandra L., Jumadilova, Zhanna, Ciesielska, Monika, Konstan, Michael W.

Source: In Journal of Cystic Fibrosis November 2021 20(6):1010-1017

A Longitudinal Study of Glucose Tolerance in Cystic Fibrosis: The Central Role of Beta Cell Functional Mass

Authors: Piona, Claudia, Mozzillo, Enza, Tosco, Antonella, Zusi, Chiara, Emiliani, Federica, Volpi, Sonia, Di Candia, Francesca, Raia, Valeria, Boselli, Maria Linda, Trombetta, Maddalena, Cipolli, Marco, Bonadonna, Riccardo C, Maffeis, Claudio

Source: The Journal of Clinical Endocrinology & Metabolism; May 2025, Vol. 110 Issue: 5 pe1670-e1677, 8p

Vanzacaftor–tezacaftor–deutivacaftor versus elexacaftor–tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials

Authors: Keating, Claire, Yonker, Lael M, Vermeulen, François, Prais, Dario, Linnemann, Rachel W, Trimble, Aaron, Kotsimbos, Tom, Mermis, Joel, Braun, Andrew T, O'Carroll, Mark, Sutharsan, Sivagurunathan, Ramsey, Bonnie, Mall, Marcus A, Taylor-Cousar, Jennifer L, McKone, Edward F, Tullis, Elizabeth, Floreth, Tim, Michelson, Peter, Sosnay, Patrick R, Nair, Nitin, Zahigian, Rachel, Martin, Hannah, Ahluwalia, Neil, Lam, Anna, Horsley, Alexander, Krick, Stefanie, McCoy, Karen, Goss, Christopher, Weiner, Daniel, Billings, Joanne, Ren, Clement, Atkinson, Jeffrey, Linnemann, Rachel, Trimble, Aaron, Frederick, Carla, Liou, Theodore, Escobar, Hugo, Wang, Janice, Deschamps, Ashley, Froh, Deborah, Chmiel, James, Wurth, Mark, Yonker, Lael, Jain, Manu, Millard, Susan, Tolle, James, Filbrun, Amy, Taylor-Cousar, Jennifer, Ruiz, Fadel, Brown, Cynthia, Quick, Bryon, Brown, Perry, Kennedy, John, Ruddy, Jennifer, Shkolnik, Boris, Pancham, Krishna, DiMango, Emily, Fiel, Stanley, Mueller, Gary, Livingston, Floyd, Braun, Andrew, O'Sullivan, Brian, Dubin, Patricia, Teneback, Charlotte, Kremer, Ted, Welter, John, Hoppe, Jordana, Jain, Raksha, Calimano, Francisco, Johannes, Jimmy, Daines, Cori, Green, Deanna, Keens, Thomas, Trillo-Alvarez, Cesar, Scher, Herschel, Mermis, Joel, Morrissey, Brian, Wallace, James, Barrios, Christopher, Callison, John, Holliday, Zachary, Klingsberg, Ross, Efrati, Ori, Cohen-Cymberknoh, Malena, Prais, Dario, Azevedo, Pilar, Quintana-Gallego, Esther, Wong, Janice, Mondejar-Lopez, Pedro, Lopez Neyra, Alejandro, Amorim, Adelina, Sivam, Sheila, Asensio de la Cruz, Oscar, Waller, Michael, Forseen, Caralee, Marion, Chad, Dittrich, Anna-Maria, Lysinger, Jerimiah, Casserly, Brian, Luna, Carmen, Brown, Sarah, Singer, Jonathan, Schellhase, Dennis, Roberts, Jon, King, Christopher, Byrnes, Catherine, Ringshausen, Felix, Hebestreit, Helge, Lorenz, Michael, Ruppel, Renate, Drevinek, Pavel, Dillenhofer, Stefanie, Stahl, Mirjam, Gleiber, Wolfgang, Homola, Lukas, Naehrig, Susanne, Stehling, Florian, Sutharsan, Sivagurunathan, Fischer, Rainald, Poplawska, Krystyna, Haworth, Charles, Simmonds, Nicholas, Urquhart, Don, Duckers, Jamie, Carroll, Mary, Barry, Peter, Doe, Simon, Hilliard, Tom, MacGregor, Gordon, Patel, Neil, Withers, Nicholas, Peckham, Daniel, Ledson, Martin, Koser, Usma, Gartner, Silvia, Blitz Castro, Enrique, Sole Jover, Amparo, Cox, Desmond, McKone, Edward, Plant, Barry, Williamson, Michael, Elnazir, Basil, Barbosa, Telma, Cols Roig, Maria, Burr, Lucy, Henderson, Daniel, Middleton, Peter, Kotsimbos, Tom, De Monestrol, Isabelle, Gilljam, Marita, Lindberg, Ulrika, Schwarz, Carsten, Bode, Sebastian, Epton, Michael, Halasz, Adrien, Laki, Istvan, O'Carroll, Mark, Krick, Stefanie, McCoy, Karen, Goss, Christopher, Weiner, Daniel, Ren, Clement, Atkinson, Jeffrey, Linnemann, Rachel, Trimble, Aaron, Fortner, Christopher, Frederick, Carla, Liou, Theodore, Escobar, Hugo, Wang, Janice, Froh, Deborah, Chmiel, James, Wurth, Mark, Bilodeau, Lara, Lapey, Allen, Jain, Manu, Millard, Susan, Tolle, James, Moffett, Kathryn, Jia, Shijing, Taylor-Cousar, Jennifer, Brown, Cynthia, Brown, Perry, Uluer, Ahmet, Ruddy, Jennifer, Sears, Edmund, Pancham, Krishna, Keating, Claire, Fiel, Stanley, Mueller, Gary, Omlor, Gregory, Braun, Andrew, Mehdi, Nighat, Tupayachi Ortiz, Maria Gabriela, Teneback, Charlotte, Kremer, Ted, Messore, Barbara, Hoppe, Jordana, Jain, Raksha, Lambert, Allison, Mukadam, Zubin, Calimano, Francisco, Daines, Cori, Egan, Marie, Green, Deanna, Keens, Thomas, Wilhelm, Andrew, Morrissey, Brian, Reyes, Santiago, Schaeffer, David, Wallace, James, Callison, John, Klingsberg, Ross, Szepfalusi, Zsolt, Epaud, Ralph, Leroy, Sylvie, Fischer Biner, Reta, Moeller, Alexander, Salvatore, Donatello, Efrati, Ori, Cohen-Cymberknoh, Malena, Prais, Dario, Johnson, Larry, Wong, Janice, Gohy, Sophie, Gramegna, Andrea, Olesen, Hanne, Tissot, Adrien, Marguet, Christophe, Sivam, Sheila, Desai, Maya, Waller, Michael, Owens, Louisa, Kherani, Tamizan, Marion, Chad, Lysinger, Jerimiah, Sands, Dorota, Bakkeheim, Egil, Tullis, Elizabeth, Chilvers, Mark, Quon, Bradley, Lands, Larry, Parkins, Michael, Tremblay, Francois, Maitra, Anirban, Casserly, Brian, Staples, Heather, Elidemir, Okan, Chatziagorou, Elpis, Tirakitsoontorn, Pornchai, Grenet, Dominique, Brown, Sarah, Linnane, Barry, Nicholson, John Michael, Manika, Katerina, Tatopoulos, Aurelie, Byrnes, Catherine, Ruppel, Renate, Stahl, Mirjam, Pressler, Tacjana, Stehling, Florian, Sutharsan, Sivagurunathan, Bagheri-Potthoff, Azadeh, Fischer, Rainald, Haworth, Charles, Simmonds, Nicholas, Duckers, Jamie, Legg, Julian, Horsley, Alexander, MacGregor, Gordon, Peckham, Daniel, Lee, Timothy, Houdouin, Veronique, Ramel, Sophie, Fajac, Isabelle, Sermet-Gaudelus, Isabelle, Kamphuis, Lieke, van der Ent, Kors, Vermeulen, Francois, Chiron, Raphael, Reix, Philippe, Van Braeckel, Eva, Knoop, Christiane, Vanderhelst, Eef, Durieu, Isabelle, Bui, Stephanie, Weiss, Laurence, Le Rouzic, Olivier, van der Meer, Renske, Roukema, Jolt, Altenburg, Josje, Cox, Desmond, McKone, Edward, Plant, Barry, Williamson, Michael, Robinson, Philip, Wainwright, Claire, Burr, Lucy, Henderson, Daniel, Mulrennan, Siobhain, Clements, Barry, Middleton, Peter, Kotsimbos, Tom, Taccetti, Giovanni, Cipolli, Marco, Dacco, Valeria, Fabrizzi, Benedetta, Castellani, Carlo, Eber, Ernst, Appelt, Dorothea, Studnicka, Michael, De Monestrol, Isabelle, Gilljam, Marita, Sapiejka, Ewa, Mazurek, Henryk, Lindberg, Ulrika, Engan, Mette, Sauty, Alain, Mankikian, Julie, Epton, Michael, Halasz, Adrien, Laki, Istvan, O'Carroll, Mark

Source: The Lancet Respiratory Medicine; March 2025, Vol. 13 Issue: 3 p256-271, 16p

Shwachman-Diamond Syndrome and Diabetes: An Update from the Italian Registry and Review of the Literature.

Authors: Minelli, Antonella, Pintani, Emily, Valli, Roberto, Tridello, Gloria, Porta, Giovanni, Fioredda, Francesca, Cipolli, Marco, Danesino, Cesare

Source: Experimental & Clinical Endocrinology & Diabetes; Feb2025, Vol. 133 Issue 2, p78-82, 5p

Subject Terms: TYPE 2 diabetes, EXOCRINE pancreatic insufficiency, TYPE 1 diabetes, GLUCOSE tolerance tests, BONE marrow

Method of treatment of Shwachman-diamond syndrome

Occurrence, outcomes and predictors of portal hypertension in cystic fibrosis: A longitudinal prospective birth cohort study

Authors: Cipolli, Marco, Fethney, Judith, Waters, Donna, Zanolla, Luisa, Meneghelli, Ilaria, Dutt, Shoma, Assael, Baroukh Maurice, Gaskin, Kevin John

Source: In Journal of Cystic Fibrosis May 2020 19(3):455-459

A Prospective Study of Hematologic Complications and Long-Term Survival of Italian Patients Affected by Shwachman-Diamond Syndrome

Authors: Cesaro, Simone, Pegoraro, Anna, Sainati, Laura, Lucidi, Vincenzina, Montemitro, Enza, Corti, Paola, Ramenghi, Ugo, Nasi, Cristina, Menna, Giuseppe, Zecca, Marco, Danesino, Cesare, Nicolis, Elena, Pasquali, Francesco, Perobelli, Sandra, Tridello, Gloria, Farruggia, Piero, Cipolli, Marco

Source: In The Journal of Pediatrics April 2020 219:196-201

Amikacin liposome inhalation suspension for chronic Pseudomonas aeruginosa infection in cystic fibrosis

Authors: Renner, Sabine, Knoop, Christiane, Malfroot, Anne, Dupont, Lieven, Desager, Kristine, De Baets, Frans, Bosheva, Miroslava, Nedkova, Vania, Galabov, Ivan, Galeva, Ivanka, Freitag, Andreas, Morrison, Nancy, Wilcox, Pearce, Pressler, Tanja, Martinet, Yves, Chiron, Raphael, Fajac, Isabelle, Dominique, Stephan, Reix, Philippe, Prevotat, Anne, Sermet, Isabelle, Durieu, Isabelle, Fischer, Rainald, Huber, Rudolf, Staab, Doris, Mellies, Uwe, Sextro, Wolfgang, Welte, Tobias, Wilkens, Heinrike, Sommerwerk, Urte, Bewig, Burkhard, Inglezos, Ilias, Doudounakis, Stavros-Eleftherios, Bede, Olga, Gönczi, Ferenc, Újhelyi, Rita, McKone, Edward, McNally, Paul, Lucidi, Vincenzina, Cipolli, Marco, La Rosa, Mario, Minicucci, Laura, Padoan, Rita, Pisi, Giovanna, Gagliardini, Rolando, Colombo, Carla, Bronsveld, Inez, Sapiejka, Ewa, Mazurek, Henryk, Sands, Dorota, Górnicka, Grażyna, Stelmach, Iwona, Batura-Gabryel, Halina, Rachel, Marta, Minic, Predrag, Orosova, Jaroslava, Takac, Branko, Feketova, Anna, Martinez, Carmen, Hernandez, Gloria Garcia, Villa-Asensi, Jose Ramon, Gartner, Silvia, Sole, Amparo, Lindblad, Anders, Ledson, Martin, Bilton, Diana, Whitehouse, Joanna, Smyth, Alan, Ketchell, Ian, Lee, Timothy, MacGregor, Gordon, Pressler, Tacjana, Clancy, John Paul, Solé, Amparo, Quittner, Alexandra L., Liu, Keith, McGinnis, John P., II, Eagle, Gina, Gupta, Renu, Konstan, Michael W.

Source: In Journal of Cystic Fibrosis March 2020 19(2):284-291

Antisense oligonucleotide eluforsen is safe and improves respiratory symptoms in F508DEL cystic fibrosis

Authors: Drevinek, Pavel, Pressler, Tacjana, Cipolli, Marco, De Boeck, Kris, Schwarz, Carsten, Bouisset, Florilene, Boff, Marie, Henig, Noreen, Paquette-Lamontagne, Nicolas, Montgomery, Sonya, Perquin, Jaakko, Tomkinson, Nigel, den Hollander, Wilhelmina, Elborn, J. Stuart

Source: In Journal of Cystic Fibrosis January 2020 19(1):99-107

Real-Life Experience with CFTR Modulators Shows Correction of LAD-IV Phenotype in Cystic Fibrosis.

Authors: Montresor, Alessio, D'Ulivo, Beatrice, Preato, Sara, Farinazzo, Alessia, Pintani, Emily, Chiara, Elena Dalla, Torroni, Lorena, Verlato, Giuseppe, Boscia, Silvia, Pisano, Laura, Mangone, Giusi, Ricci, Silvia, Azzari, Chiara, Taccetti, Giovanni, Terlizzi, Vito, Cipolli, Marco, Melotti, Paola, Sorio, Claudio, Laudanna, Carlo

Source: American Journal of Respiratory Cell & Molecular Biology; Oct2024, Vol. 71 Issue 4, p495-498, 4p

Subject Terms: DRUG accessibility, RHO GTPases, BONE marrow, CYSTIC fibrosis, CYSTIC fibrosis transmembrane conductance regulator, CHLORIDE channels, MUCOCILIARY system