Alpelisib for PIK3CA-Mutated, Hormone Receptor–Positive Advanced Breast Cancer von André, Fabrice, Ciruelos, Eva, Rubovszky, Gabor, Campone, Mario, Loibl, Sibylle, Rugo, Hope S, Iwata, Hiroji, Conte, Pierfranco, Mayer, Ingrid A, Kaufman, Bella, Yamashita, Toshinari, Lu, Yen-Shen, Inoue, Kenichi, Takahashi, Masato, Pápai, Zsuzsanna, Longin, Anne-Sophie, Mills, David, Wilke, Celine, Hirawat, Samit, Juric, Dejan

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Ribociclib as First-Line Therapy for HR-Positive, Advanced Breast Cancer von Hortobagyi, Gabriel N, Stemmer, Salomon M, Burris, Howard A, Yap, Yoon-Sim, Sonke, Gabe S, Paluch-Shimon, Shani, Campone, Mario, Blackwell, Kimberly L, André, Fabrice, Winer, Eric P, Janni, Wolfgang, Verma, Sunil, Conte, Pierfranco, Arteaga, Carlos L, Cameron, David A, Petrakova, Katarina, Hart, Lowell L, Villanueva, Cristian, Chan, Arlene, Jakobsen, Erik, Nusch, Arnd, Burdaeva, Olga, Grischke, Eva-Maria, Alba, Emilio, Wist, Erik, Marschner, Norbert, Favret, Anne M, Yardley, Denise, Bachelot, Thomas, Tseng, Ling-Ming, Blau, Sibel, Xuan, Fengjuan, Souami, Farida, Miller, Michelle, Germa, Caroline, Hirawat, Samit, O’Shaughnessy, Joyce

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Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial von Kerem, Eitan, Dr, Hirawat, Samit, MD, Armoni, Shoshana, BSN, Yaakov, Yasmin, MSc, Shoseyov, David, MD, Cohen, Michael, MD, Nissim-Rafinia, Malka, PhD, Blau, Hannah, MD, Rivlin, Joseph, MD, Aviram, Micha, MD, Elfring, Gary L, MS, Northcutt, Valerie J, MS, Miller, Langdon L, MD, Kerem, Batsheva, PhD, Wilschanski, Michael, MBBS

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Targeting activated PI3K/mTOR signaling overcomes acquired resistance to CDK4/6-based therapies in preclinical models of hormone receptor-positive breast cancer von O'Brien, Neil A, McDermott, Martina S J, Conklin, Dylan, Luo, Tong, Ayala, Raul, Salgar, Suruchi, Chau, Kevin, DiTomaso, Emmanuelle, Babbar, Naveen, Su, Faye, Gaither, Alex, Hurvitz, Sara A, Linnartz, Ronald, Rose, Kristine, Hirawat, Samit, Slamon, Dennis J

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Buparlisib and paclitaxel in patients with platinum-pretreated recurrent or metastatic squamous cell carcinoma of the head and neck (BERIL-1): a randomised, double-blind, placebo-c... von Soulières, Denis, Prof, Faivre, Sandrine, Prof, Mesía, Ricard, Prof, Remenár, Éva, Prof, Li, Shau-Hsuan, Prof, Karpenko, Andrey, Prof, Dechaphunkul, Arunee, MD, Ochsenreither, Sebastian, Prof, Kiss, Laura Anna, Prof, Lin, Jin-Ching, Prof, Nagarkar, Raj, Prof, Tamás, László, Prof, Kim, Sung-Bae, Prof, Erfán, Jozsef, Prof, Alyasova, Anna, Prof, Kasper, Stefan, Prof, Barone, Carlo, Prof, Turri, Sabine, MS, Chakravartty, Arunava, PhD, Chol, Marie, PhD, Aimone, Paola, Prof, Hirawat, Samit, Prof, Licitra, Lisa, Prof

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PI3K inhibitors as new cancer therapeutics: implications for clinical trial design von Massacesi, Cristian, Di Tomaso, Emmanuelle, Urban, Patrick, Germa, Caroline, Quadt, Cornelia, Trandafir, Lucia, Aimone, Paola, Fretault, Nathalie, Dharan, Bharani, Tavorath, Ranjana, Hirawat, Samit

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PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model von Du, Ming, Liu, Xiaoli, Welch, Ellen M, Hirawat, Samit, Peltz, Stuart W, Bedwell, David M

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The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy von McDonald, Craig M., Henricson, Erik K., Han, Jay J., Abresch, R. Ted, Nicorici, Alina, Elfring, Gary L., Atkinson, Leone, Reha, Allen, Hirawat, Samit, Miller, Langdon L.

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Targeting PI3K/mTOR overcomes resistance to HER2-targeted therapy independent of feedback activation of AKT von O'Brien, Neil A, McDonald, Karen, Tong, Luo, von Euw, Erika, Kalous, Ondrej, Conklin, Dylan, Hurvitz, Sara A, di Tomaso, Emmanuelle, Schnell, Christian, Linnartz, Ronald, Finn, Richard S, Hirawat, Samit, Slamon, Dennis J

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Challenges in the clinical development of PI3K inhibitors von Massacesi, Cristian, di Tomaso, Emmanuelle, Fretault, Nathalie, Hirawat, Samit

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Safety, Tolerability, and Pharmacokinetics of PTC124, a Nonaminoglycoside Nonsense Mutation Suppressor, Following Single- and Multiple-Dose Administration to Healthy Male and Femal... von Hirawat, Samit, Welch, Ellen M., Elfring, Gary L., Northcutt, Valerie J., Paushkin, Sergey, Hwang, Seongwoo, Leonard, Eileen M., Almstead, Neil G., Ju, William, Peltz, Stuart W., Miller, Langdon L.

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Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation von Weetall, Marla, Davis, Thomas, Elfring, Gary, Northcutt, Valerie, Cao, Liangxian, Moon, Young-Choon, Riebling, Peter, Dali, Mandar, Hirawat, Samit, Babiak, John, Colacino, Joseph, Almstead, Neil, Spiegel, Robert, Peltz, Stuart W.

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Ribociclib plus endocrine therapy for premenopausal women with hormone-receptor-positive, advanced breast cancer (MONALEESA-7): a randomised phase 3 trial von Tripathy, Debu, Im, Seock-Ah, Colleoni, Marco, Franke, Fabio, Bardia, Aditya, Harbeck, Nadia, Hurvitz, Sara A, Chow, Louis, Sohn, Joohyuk, Lee, Keun Seok, Campos-Gomez, Saul, Villanueva Vazquez, Rafael, Jung, Kyung Hae, Babu, K Govind, Wheatley-Price, Paul, De Laurentiis, Michelino, Im, Young-Hyuck, Kuemmel, Sherko, El-Saghir, Nagi, Liu, Mei-Ching, Carlson, Gary, Hughes, Gareth, Diaz-Padilla, Ivan, Germa, Caroline, Hirawat, Samit, Lu, Yen-Shen

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Phase Ib Study of Buparlisib plus Trastuzumab in Patients with HER2-Positive Advanced or Metastatic Breast Cancer That Has Progressed on Trastuzumab-Based Therapy von SAURA, Cristina, BENDELL, Johanna, BECK, Joseph T, DI TOMASO, Emmanuelle, STERNBERG, David W, MASSACESI, Cristian, HIRAWAT, Samit, DIRIX, Luc, BASELGA, Jose, JERUSALEM, Guy, SHAUN SU, QINHUA RU, DE BUCK, Stefan, MILLS, David, RUQUET, Sophie, BOSCH, Ana, URRUTICOECHEA, Ander

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Ataluren (PTC124) Induces Cystic Fibrosis Transmembrane Conductance Regulator Protein Expression and Activity in Children with Nonsense Mutation Cystic Fibrosis von SERMET-GAUDELUS, Isabelle, DE BOECK, Kris, MILLERS, Nilsen L, CONSTANTINE, Scott, REHA, Allen, AJAYI, Temitayo, ELFRING, Gary L, MILLERS, Langdon L, CASIMIR, Georges J, VERMEULEN, François, LEAL, Teresinha, MOGENET, Agnès, ROUSSEL, Delphine, FRITSCH, Janine, HANSSENS, Laurence, HIRAWAT, Samit

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PTC124 targets genetic disorders caused by nonsense mutations von Chen, Guangming, Jacobson, Allan, Spatrick, Phyllis, Peltz, Stuart W, Conn, M. Morgan, Friesen, Westley J, Almstead, Neil G, Hedrick, Jean, Campbell, Jeffrey A, Mollin, Anna, Kawana, Masataka, Wilde, Richard G, Yeh, Shirley, Sweeney, H. Lee, Trotta, Christopher R, Khan, Atiyya, Welch, Ellen M, Barton, Elisabeth R, Hirawat, Samit, Turpoff, Anthony A, Weetall, Marla, Ren, Hongyu, Risher, Nicole, Tomizawa, Yuki, Northcutt, Valerie J, Miller, Langdon L, He, Feng, Jones, Stephen, Colacino, Joseph M, Feng, Huisheng, Branstrom, Arthur A, Corson, Donald, Zhuo, Jin, Ju, William D, Patel, Meenal, Moon, Young-Choon, Hwang, Seongwoo, Paushkin, Sergey, Babiak, John, Trifillis, Panayiota, Karp, Gary, Takasugi, James

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Abstract 3825: Targeting activated PI3K/mTOR signaling overcomes resistance to CDK4/6-based therapies in preclinical ER+ breast cancer models von O'Brien, Neil A., McDermott, Martina SJ, Conklin, Dylan F., Gaither, Alex, Luo, Tong, Ayala, Raul, Salgar, Suruchi, DiTomaso, Emmanuelle, Babbar, Naveen, Su, Faye, Hurvitz, Sara A., Linnartz, Ronald, Rose, Kristine, Hirawat, Samit, Slamon, Dennis J.

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Abstract 4698: Combination of ceritinib (LDK378) with PI3K inhibitors (buparlisib [BKM120] and alpelisib [BYL719]) demonstrates synergistic preclinical antitumor activity in ALK-re... von Di Tomaso, Emmanuelle, Linnartz, Ronald, Li, Fang, Massacesi, Cristian, Hirawat, Samit

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Some practical considerations for phase III studies with biomarker evaluations von Goteti, Sasikiran, Hirawat, Samit, Massacesi, Cristian, Fretault, Nathalie, Bretz, Frank, Dharan, Bharani

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BERIL-1: Biomarker results from targeted sequencing of circulating tumor DNA (ctDNA) and archival tissue in a randomized phase II study of buparlisib (BKM120) or placebo plus pacli... von Licitra, Lisa F., Faivre, Sandrine J., Chol, Marie, Solovieff, Nadia, Robinson, Douglas, DiTomaso, Emmanuelle, Hirawat, Samit, Soulieres, Denis

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