Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis von WILSCHANSKI, M, MILLER, L. L, COHEN-CYMBERKNOH, M, MILLER, N. L, REHA, A, NORTHCUTT, V. J, HIRAWAT, S, DONNELLY, K, ELFRING, G. L, AJAYI, T, KEREM, E, SHOSEYOV, D, BLAU, H, RIVLIN, J, AVIRAM, M, COHEN, M, ARMONI, S, YAAKOV, Y, PUGATCH, T

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Phase II trial of irinotecan in patients with progressive or rapidly recurrent colorectal cancer von ROTHENBERG, M. L, ECKARDT, J. R, WEISS, G. R, ELFRING, G. L, RINALDI, D. A, SCHAAF, L. J, VAN HOFF, D. D, KUHN, J. G, BURRIS, H. A, NELSON, J, HILSENBECK, S. G, RODRIGUEZ, G. I, THURMAN, A. M, SMITH, L. S, ECKHARDT, S. G

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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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Irinotecan Therapy in Adults With Recurrent or Progressive Malignant Glioma von FRIEDMAN, H. S, PETROS, W. P, CLOUGHSEY, T, STEWART, E. S, COLVIN, O. M, PROVENZALE, J. M, MCLENDON, R. E, BIGNER, D. D, COKGOR, L, HAGLUND, M, RICH, J, ASHLEY, D, FRIEDMAN, A. H, MALCZYN, J, ELFRING, G. L, MILLER, L. L, SCHAAF, L. J, KERBY, T, LAWYER, J, PARRY, M, HOUGHTON, P. J, LOVELL, S, RASHEED, K

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Phase I dose-finding and pharmacokinetic trial of irinotecan (CPT-11) administered every two weeks von Rothenberg, M. L., Kuhn, J. G., Schaaf, L. J., Rodriguez, G.I., Eckhardt, S. G., Villalona-Calero, M. A., Rinaldi, D. A., Hammond, L. A., Hodges, S., Sharma, A., Elfring, G. L., Petit, R. G., Locker, P. K., Miller, L. L., von Hoff, D. D.

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Phase I and Pharmacokinetic Trial of Oral Irinotecan Administered Daily for 5 Days Every 3 Weeks in Patients With Solid Tumors von DRENGLER, R. L, KUHN, J. G, ELFRING, G. L, LOCKER, P. K, MILLER, L. L, VON HOFF, D. D, ROTHENBERG, M. L, SCHAAF, L. J, RODRIGUEZ, G. I, VILLALONA-CALERO, M. A, HAMMOND, L. A, STEPHENSON, J. A, HODGES, S, KRAYNAK, M. A, STATON, B. A

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Phase II Study of Irinotecan Plus Cisplatin in Patients With Advanced Non–Small-Cell Lung Cancer von DEVORE, R. F, JOHNSON, D. H, MILLER, L. L, CRAWFORD, J, GARST, J, DIMERY, I. W, ECKARDT, J, ECKHARDT, S. G, ELFRING, G. L, SCHAAF, L. J, HANOVER, C. K

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Phase I clinical and pharmacologic study of weekly cisplatin combined with weekly irinotecan in patients with advanced solid tumors von SALTZ, L. B, SPRIGGS, D, SEMPLE, D, HANOVER, C. K, ELFRING, G. L, MILLER, L. L, KELSEN, D, SCHAAF, L. J, SCHWARTZ, G. K, ILSON, D, KEMENY, N, KANOWITZ, J, STEGER, C, ENG, M, ALBANESE, P

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Two studies evaluating irinotecan treatment for recurrent malignant glioma using an every‐3‐week regimen von Cloughesy, Timothy F., Filka, Emese, Kuhn, John, Nelson, Gillian, Kabbinavar, Fairooz, Friedman, Henry, Miller, Langdon L., Elfring, Gary L.

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Phase I Trial of the Combination of Irinotecan, Paclitaxel, and Carboplatin in Patients With Advanced Non–Small-Cell Lung Cancer von SOCINSKI, Mark A, SANDLER, Alan B, MILLER, Langdon L, LOCKER, Paula K, HANOVER, Cristy K, ELFRING, Gary L, ISRAEL, Valerie K, PIROTTA, Nicoletta, NATALE, Ronald B

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Irinotecan Plus Fluorouracil/Leucovorin for Metastatic Colorectal Cancer: A New Survival Standard von Saltz, Leonard B., Douillard, Jean‐Yves, Pirotta, Nicoletta, Alakl, May, Gruia, Gabriela, Awad, Lucile, Elfring, Gary L., Locker, Paula K., Miller, Langdon L.

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Ataluren treatment of patients with nonsense mutation dystrophinopathy von Bushby, Katharine, Finkel, Richard, Wong, Brenda, Barohn, Richard, Campbell, Craig, Comi, Giacomo P., Connolly, Anne M., Day, John W., Flanigan, Kevin M., Goemans, Nathalie, Jones, Kristi J., Mercuri, Eugenio, Quinlivan, Ros, Renfroe, James B., Russman, Barry, Ryan, Monique M., Tulinius, Mar, Voit, Thomas, Moore, Steven A., Lee Sweeney, H., Abresch, Richard T., Coleman, Kim L., Eagle, Michelle, Florence, Julaine, Gappmaier, Eduard, Glanzman, Allan M., Henricson, Erik, Barth, Jay, Elfring, Gary L., Reha, Allen, Spiegel, Robert J., O'donnell, Michael W., Peltz, Stuart W., Mcdonald, Craig M.

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Irinotecan plus Fluorouracil and Leucovorin for Metastatic Colorectal Cancer von Saltz, Leonard B, Cox, John V, Blanke, Charles, Rosen, Lee S, Fehrenbacher, Louis, Moore, Malcolm J, Maroun, Jean A, Ackland, Stephen P, Locker, Paula K, Pirotta, Nicoletta, Elfring, Gary L, Miller, Langdon L

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Phase I study of adozelesin (U-73,975) in patients with solid tumors von Shamdas, Glenn J, Alberts, David S, Modlano, Manuel, Wiggins, Cindy, Power, Jane, Kasunic, Dorothy A, Elfring, Gary L, Earhart, Robert H

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P4.10 The relationship of ataluren plasma concentration and response across clinical studies in nonsense mutation dystrophinopathy von Finkel, R, Wong, B, Bushby, K, Voit, T, Morsy, M, Elfring, G.L, Barth, J, Peltz, S.W

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Results of a study of the oral inhibitor PTC299 on tumor VEGF synthesis von Luke, J. J., Callahan, L. A., Darby, C. H., Moss, E., Winkelman, J., Barth, J., Elfring, G. L., Ogden, A., Schwartz, G. K., Gounder, M. M.

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Reduction of serum VEGF and IL-6 levels in patients with metastatic breast cancer: Results of a study of PTC299, an oral inhibitor of tumor VEGF synthesis, and aromatase inhibitors von Dickler, M. N., Schneider, B. P., Volm, M., Speyer, J. L., Novik, Y., Callahan, L. A., Darby, C. H., Barth, J., Elfring, G. L., Ogden, A., Tiersten, A.

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P1.33 Disease progression observed in clinical outcome measures in placebo-treated patients with nonsense mutation dystrophinopathy von Mercuri, E, Bushby, K, McDonald, C, Goemans, N, Muntoni, F, Darras, B.T, Elfring, G.L, Barth, J, Reha, A, Peltz, S.W

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PP3.3 – 1808 Disease-related symptoms and activities of daily living: a novel survey of patients with nonsense mutation Duchenne muscular dystrophy von Reha, A, Barth, J, Elfring, GL, Spiegel, R

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Irinotecan Plus Gemcitabine Induces Both Radiographic and CA 19-9 Tumor Marker Responses in Patients With Previously Untreated Advanced Pancreatic Cancer von Rocha Lima, C. M. S.

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