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

Role of viable but non culturable cells in patients with cystic fibrosis in the era of highly effective modulator therapy

Authors: Cirilli, Natalia, Schiavoni, Valentina, Tagliabracci, Valentina, Gesuita, Rosaria, Tiano, Luca, Fabrizzi, Benedetta, D'Antuono, Anastasia, Peruzzi, Arianna, Cedraro, Nicholas, Carle, Flavia, Moretti, Marco, Ferrante, Luigi, Vignaroli, Carla, Biavasco, Francesca, Mangiaterra, Gianmarco

Source: In Journal of Cystic Fibrosis November 2024 23(6):1153-1158

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

Different management approaches and outcome for infants with an inconclusive diagnosis following newborn screening for cystic fibrosis (CRMS/CFSPID) and Pseudomonas aeruginosa isolation

Authors: Dolce, Daniela, Claut, Laura, Colombo, Carla, Tosco, Antonella, Castaldo, Alice, Padoan, Rita, Timpano, Silviana, Fabrizzi, Benedetta, Bonomi, Paolo, Taccetti, Giovanni, Terlizzi, Vito

Source: In Journal of Cystic Fibrosis January 2023 22(1):73-78

Clinical outcomes of a large cohort of individuals with the F508del/5T;TG12 CFTR genotype

Authors: Tosco, Antonella, Castaldo, Alice, Colombo, Carla, Claut, Laura, Carnovale, Vincenzo, Iacotucci, Paola, Lucarelli, Marco, Cimino, Giuseppe, Fabrizzi, Benedetta, Caporelli, Nicole, Majo, Fabio, Ciciriello, Fabiana, Padoan, Rita, Poli, Piercarlo, Taccetti, Giovanni, Centrone, Claudia, Casciaro, Rosaria, Castellani, Carlo, Salvatore, Donatello, Colangelo, Carmela, Bonomi, Paolo, Castaldo, Giuseppe, Terlizzi, Vito

Source: In Journal of Cystic Fibrosis September 2022 21(5):850-855

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

A survey of the prevalence, management and outcome of infants with an inconclusive diagnosis following newborn bloodspot screening for cystic fibrosis (CRMS/CFSPID) in six Italian centres

Authors: Terlizzi, Vito, Claut, Laura, Tosco, Antonella, Colombo, Carla, Raia, Valeria, Fabrizzi, Benedetta, Lucarelli, Marco, Angeloni, Antonio, Cimino, Giuseppe, Castaldo, Alice, Marsiglio, Laura, Timpano, Silviana, Cirilli, Natalia, Moroni, Laura, Festini, Filippo, Piccinini, Pietro, Zavataro, Lucia, Bonomi, Paolo, Taccetti, Giovanni, Southern, Kevin W, Padoan, Rita

Source: In Journal of Cystic Fibrosis September 2021 20(5):828-834

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

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

Authors: 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, Wainwright, Claire E

Source: In The Lancet Respiratory Medicine July 2021 9(7):733-746

Critical Issues in the Management of CRMS/CFSPID Children: A National Real‐World Survey.

Authors: Terlizzi, Vito, Fevola, Cristina, Presti, Santiago, Claut, Laura, Ambroni, Maura, Calderazzo, Maria Adelaide, Esposito, Irene, Fabrizzi, Benedetta, Leonetti, Giuseppina, Lombardo, Mariangela, Maschio, Massimo, Palladino, Nicola, Pauro, Francesca, Pisi, Giovanna, Ripani, Pietro, Ros, Mirco, Rotolo, Novella, Salvatore, Donatello, Sepe, Angela, Termini, Lisa

Source: Pediatric Pulmonology; Jan2025, Vol. 60 Issue 1, p1-6, 6p

Clinical relevance of Pseudomonas aeruginosa viable but non-culturable forms in cystic fibrosis.

Authors: Mangiaterra, Gianmarco, Schiavoni, Valentina, Cedraro, Nicholas, Citterio, Barbara, Vignaroli, Carla, Gesuita, Rosaria, Fabrizzi, Benedetta, Biavasco, Francesca, Cirilli, Natalia

Source: European Journal of Clinical Microbiology & Infectious Diseases; Sep2024, Vol. 43 Issue 9, p1865-1867, 3p

Subject Terms: CYSTIC fibrosis transmembrane conductance regulator, CYSTIC fibrosis, DISEASE relapse, PSEUDOMONAS aeruginosa, INTRAVENOUS injections

A Multicentre Italian Study on the Psychological Impact of an Inconclusive Cystic Fibrosis Diagnosis after Positive Neonatal Screening.

Authors: Tosco, Antonella, Marino, Diletta, Polizzi, Sara, Tradati, Valentina, Padoan, Rita, Giust, Claudia, Fabrizzi, Benedetta, Taccetti, Giovanni, Merli, Lucia, Terlizzi, Vito

Source: Children; Feb2023, Vol. 10 Issue 2, p177, 11p

Subject Terms: NEWBORN screening, RESEARCH, CYSTIC fibrosis, RESEARCH funding

Geographic Terms: ITALY

Role of Bronchial Artery Embolization as Early Treatment Option in Stable Cystic Fibrosis Patients with Sub-Massive Hemoptysis: Personal Experience and Literature Review.

Authors: Floridi, Chiara, Boscarato, Pietro, Ventura, Claudio, Bruno, Alessandra, Rossini, Nicolo', Baldassari, Michela, Lanza, Cecilia, Fabrizzi, Benedetta, Candelari, Roberto, Giovagnoni, Andrea

Source: Journal of Clinical Medicine; Nov2022, Vol. 11 Issue 21, p6432, 11p

Subject Terms: BRONCHIAL arteries, CYSTIC fibrosis, LIFE expectancy, ARTERIAL catheterization, HEMOPTYSIS

Clinical outcome of individuals carrying 5T;TG12 in trans with CFTR variants with varying clinical consequences.

Authors: Tosco, Antonella, Carnovale, Vincenzo, Claut, Laura, Fabrizzi, Benedetta, Majo, Fabio, Castellani, Carlo, Sepe, Angela, Castaldo, Giuseppe, Terlizzi, Vito

Source: Pediatric Pulmonology; Apr2023, Vol. 58 Issue 4, p1253-1255, 3p

Outcomes of early repeat sweat testing in infants with cystic fibrosis transmembrane conductance regulator‐related metabolic syndrome/CF screen‐positive, inconclusive diagnosis.

Authors: Terlizzi, Vito, Claut, Laura, Colombo, Carla, Tosco, Antonella, Castaldo, Alice, Fabrizzi, Benedetta, Lucarelli, Marco, Cimino, Giuseppe, Carducci, Carla, Dolce, Daniela, Biffi, Arianna, Bonomi, Paolo, Timpano, Silviana, Padoan, Rita

Source: Pediatric Pulmonology; Dec2021, Vol. 56 Issue 12, p3785-3791, 7p

Two novel and correlated CF-causing insertions in the (TG)mTn tract of the CFTR gene.

Authors: Pierandrei, Silvia, Blaconà, Giovanna, Fabrizzi, Benedetta, Cimino, Giuseppe, Cirilli, Natalia, Caporelli, Nicole, Angeloni, Antonio, Cipolli, Marco, Lucarelli, Marco

Source: PLoS ONE; 10/7/2019, Vol. 14 Issue 10, p1-14, 14p

Subject Terms: CYSTIC fibrosis transmembrane conductance regulator, BURKHOLDERIA infections, NON-coding RNA, RNA splicing, MEDICAL genetics

Breakthroughs in Preclinical Development of Ataluren (PTC124) As Therapeutic Option for Patients Affected By Shwachman-Diamond Syndrome: Towards the First Clinical Trial

Authors: Bezzerri, Valentino ^*, Vella, Antonio ^*, D'Aversa, Elisabetta ^*, Api, Martina ^*, Allegri, Marisole ^*, Marinelli Busilacchi, Elena ^*, D'Amico, Giovanna, Gambari, Roberto ^*, Sorio, Claudio ^*, Fabrizzi, Benedetta ^*, Bronte, Vincenzo ^*, Poloni, Antonella ^*, Borgatti, Monica ^*, Cipolli, Marco ^*

Source: In Blood 13 November 2019 134 Supplement 1:451-451

CRMS/CFSPID Subjects Carrying D1152H CFTR Variant: Can the Second Variant Be a Predictor of Disease Development?

Authors: Terlizzi, Vito, Padoan, Rita, Claut, Laura, Colombo, Carla, Fabrizzi, Benedetta, Lucarelli, Marco, Bruno, Sabina Maria, Castaldo, Alice, Bonomi, Paolo, Taccetti, Giovanni, Tosco, Antonella

Source: Diagnostics (2075-4418); Dec2020, Vol. 10 Issue 12, p1080-1080, 1p

Subject Terms: TREATMENT effectiveness, CYSTIC fibrosis, DIAGNOSIS, PSEUDOMONAS aeruginosa

Nonsense Suppression Therapy: New Hypothesis for the Treatment of Inherited Bone Marrow Failure Syndromes.

Authors: Bezzerri, Valentino, Api, Martina, Allegri, Marisole, Fabrizzi, Benedetta, Corey, Seth J., Cipolli, Marco

Source: International Journal of Molecular Sciences; Jul2020, Vol. 21 Issue 13, p4672-4672, 1p

Subject Terms: BONE marrow, RIBOSOMES, TELOMERES, HEMATOPOIETIC stem cell transplantation, BONE marrow cells, PATHOLOGY, ORGANELLE formation

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

Authors: 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, Gilljam, Marita

Source: The Lancet Respiratory Medicine; 20210101, Issue: Preprints