Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV) von DeVincenzo, John, Cehelsky, Jeffrey E., Alvarez, Rene, Elbashir, Sayda, Harborth, Jens, Toudjarska, Iva, Nechev, Lubomir, Murugaiah, Veeravagu, Vliet, Andre Van, Vaishnaw, Akshay K., Meyers, Rachel

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Phase II trial of intravenous lobradimil and carboplatin in childhood brain tumors : a report from the Children's Oncology Group von WARREN, K, JAKACKI, R, OSBORNE, C, CEHELSKY, J, CALDWELL, D, STANWOOD, J, STEINBERG, S. M, BALIS, F. M, WIDEMANN, B, AIKIN, A, LIBUCHA, M, PACKER, R, VEZINA, G, REAMAN, G, SHAW, D, KRAILO, M

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Burden of Illness for Patients with familial amyloidotic Polyneuropathy (Fap) Begins Early and Increases with Disease Progression von Denoncourt, R.N, Adams, D, Coelho, T, Bettencourt, B.R, Plaisted, A, Amitay, O, Falzone, R, Harrop, J, White, L, De Frutos, R, Cehelsky, J, Nochur, S, Vaishnaw, A.K, Gollob, J

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41: Results of a Randomized Phase 2 Trial of ALN-RSV01, an RNAi Therapeutic, Lung Transplant (LTX) Patients Infected with Respiratory Syncytial Virus (RSV) von Zamora, M.R, Budev, M, Rolfe, M, Gottlieb, J, DeVincenzo, J, Cehelsky, J, Albert, G, Gollob, J, Nochur, S, Vaishnaw, A, Glanville, A

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Development of a Human Experimental Infection Model of Respiratory Syncytial Virus (RSV) and Evaluation of an RNA Interference (RNAi) Therapeutic for Safety and Anti-Viral Efficacy... von DeVincenzo, J, Lambkin-Williams, R, Cehelsky, J, Wilkinson, T, Obute, O, Foote, K, Nochur, S, Gilberts, A, Mann, A, Moane, E, Walsh, E, Studholme, B, Dorset, P, Oxford, J, Meyers, R, Vaishnaw, A

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PND52 - Burden of Illness for Patients with familial amyloidotic Polyneuropathy (Fap) Begins Early and Increases with Disease Progression von Denoncourt, R.N., Adams, D., Coelho, T., Bettencourt, B.R., Plaisted, A., Amitay, O., Falzone, R., Harrop, J., White, L., De Frutos, R., Cehelsky, J., Nochur, S., Vaishnaw, A.K., Gollob, J.

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Phase 2 open-label extension study (ole) of patisiran, an investigational sIRNA investigational agent for familial amyloid polyneuropathy (fap) von Coelho, T., Suhr, Ole, Conceicao, I, Waddington-Cruz, M., Schmidt, H., Buades, J., Campistol, J., Pouget, J., Berk, J., Falzone, R., White, L., Bettencourt, B., Cehelsky, J., Nochur, S., Vaishnaw, A., Gollob, J., Adams, D.

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Interim safety and pharmacodynamic results for ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement von Gollob, J., Infante, J. R., Shapiro, G., LoRusso, P., Dezube, B. J., Heymach, J., Cehelsky, J., Falzone, R., Vaishnaw, A., Burris, H. A.

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Interim results from phase ii trial of aln-ttr02, a novel RNAi therapeutic for the treatment of familial amyloidotic polyneuropathy von Adams, D., Coelho, T., Suhr, Ole, Conceicao, I., Waddington-Cruz, M., Schmidt, H., Campistol, J., Pouget, J., Buades, J., Falzone, R., Harrop, J., De Frutos, R., Butler, J., Cehelsky, J., Nochur, S., Vaishnaw, A., Gollob, J.

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Phase I dose-escalation study of ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement von Cervantes, A., Alsina, M., Tabernero, J., Infante, J. R., LoRusso, P., Shapiro, G., Paz-Ares, L. G., Falzone, R., Hill, J., Cehelsky, J., White, A., Toudjarska, I., Bumcrot, D., Meyers, R., Hinkle, G., Svrzikapa, N., Sah, D. W., Vaishnaw, A., Gollob, J., Burris, H. A.

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Abstracts7th Annual Meeting of the Oligonucleotide Therapeutics SocietyCopenhagen, DenmarkSeptember 8–10, 2011 von Aartsma-Rus, Annemieke, Karnaoek, Tatyana, I, Bestel, Leatherbury, Neil, SA, Schwabe, J, Hunziker, Rockey, William M, Spencer, David M, Giangrande, Paloma H, Tavitian, Bertrand, Kjems, Jørgen, Bolcato-Bellemin, Anne-Laure, Deglane, Gaëlle, Goldeck, M., Schuberth, C., van Kessel, Ingeborg, Hartmann, Roland K., Hall, Jonathan, Gait, Michael J, Hornung, Veit, Hartmann, Gunther, Schlee, Martin, MW, Lindholm, Veedu, Rakesh N., Harbottle, R., Kazakov, S. A., NarayanannairJayaprakash, K., Wang, Gang, Sah, Dinah W.Y., Manoharan, Muthiah, Nielsen, Niels Fisker, Kauppinen, Sakari, Elmén, Joacim, Fettes, Kevin, Sobczak, Milena, Kim, Hee Won, Svrzikapa, Nenad, Qin, June, Tracy, Mark A., Makita, Naoki, Nagahara, Shunji, Stockdale, Katie R., Hanin, Geula, Gilboa-Geffen, Adi, Soreq, Hermona, McClorey, Graham, Shibata, Atsushi, Kummer, Ursula, Nawrot, Barbara, Snead, Nicholas M., Kamiya, D., Matsui, Yuriko, Tran, Phong Lan Thao, Rayner, Bernard, Urban, Johannes, Phua, Kyle Kok Loong, Harada-Shiba, Mariko, Obika, Satoshi, Agapkina, J., Aouadi, Myriam, Guo, Chang-An, Byun, Jonghoe, Tula, Saison-Behmoaras, Galun, Eithan, M, Yamamoto, Iversen, Patrick, Kim, Sera, Komoriya, Akira, Nagatsugi, Fumi, Kusano, Shuhei, Mohri, Kohta, Bernhard, Noll, Philipp, Hadwiger, Shrewsbury, Stephen, Takahashi, Mayumi, Matsuda, Akira, Sah, D.W., Gantier, Michael P., Sczakiel, Georg, C., Coch, B., Kümmerer, S., Herberhold, G., Kochs, M., Schlee, Trujillo, Robin Deis, Edbrooke, Mark, Fitzgerald-Bocarsly, Patricia, Gibson, Neil W., Bhat, Balkrishen, Chen, Hubert, Jackson, Aimee L., A, Buchbinder, Hawkins, Philip, Cehelsky, Jeff, Gollob, Jared, Moore, Michael D., Chomchan, Pritsana, Hua, Yimin, Sahashi, Kentaro, Aarts, Marieke

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CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis von Gillmore, JD, Gane, E, Taubel, J, Kao, J, Fontana, M, Maitland, ML, Seitzer, J, O'Connell, D, Walsh, KR, Wood, K, Phillips, J, Xu, Y, Amaral, A, Boyd, AP, Cehelsky, JE, McKee, MD, Schiermeier, A, Harari, O, Murphy, A, Kyratsous, CA, Zambrowicz, B, Soltys, R, Gutstein, DE, Leonard, J, Sepp-Lorenzino, L, Lebwohl, D

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First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement von Tabernero, Josep, Shapiro, Geoffrey I, LoRusso, Patricia M, Cervantes, Andres, Schwartz, Gary K, Weiss, Glen J, Paz-Ares, Luis, Cho, Daniel C, Infante, Jeffrey R, Alsina, Maria, Gounder, Mrinal M, Falzone, Rick, Harrop, Jamie, White, Amy C Seila, Toudjarska, Iva, Bumcrot, David, Meyers, Rachel E, Hinkle, Gregory, Svrzikapa, Nenad, Hutabarat, Renta M, Clausen, Valerie A, Cehelsky, Jeffrey, Nochur, Saraswathy V, Gamba-Vitalo, Christina, Vaishnaw, Akshay K, Sah, Dinah W Y, Gollob, Jared A, Burris, 3rd, Howard A

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In vivo CRISPR/Cas9 editing of the TTR gene with NTLA-2001 in patients with transthyretin amyloidosis- dose selection considerations von Gane, Edward J., Täubel, Jörg, Pilebro, Björn, Fontana, Marianna, Kao, Justin, Maitland, Michael, Stroh, Mark, Seitzer, Jessica, Phillips, Jonathan, Wood, Kristy, Xu, Yuanxin, Boiselle, Carri, Amaral, Adam, Boyd, Adam, Cehelsky, Jeffrey, Gustein, David, Pagovich, Odelya, Sepp-Lorenzino, Laura, Walsh, Liron, Lebwohl, David, Julian, Gillmore

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Open-label extension study of the RNAi therapeutic ALN-VSP02 in cancer patients responding to therapy von Alsina, Maria, Tabernero, Josep, Shapiro, Geoffrey, Burris, Howard, Infante, Jeffrey R., Weiss, Glen J., Cervantes-Ruiperez, Andres, Gounder, Mrinal M., Paz-Ares, Luis, Falzone, Rick, Hill, Jamie, Cehelsky, Jeffrey, Vaishnaw, Akshay, Gollob, Jared, LoRusso, Patricia

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Assessing Modeled CO2 Retention and Rebreathing of a Facemask Designed for Efficient Delivery of Aerosols to Infants von Mundt, Christian, Sventitskiy, Alexander, Cehelsky, Jeffrey E., Patters, Andrea B., Tservistas, Markus, Hahn, Michael C., Juhl, Gerd, DeVincenzo, John P.

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