Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression von Roy, Bijoyita, Friesen, Westley J., Tomizawa, Yuki, Leszyk, John D., Zhuo, Jin, Johnson, Briana, Dakka, Jumana, Trotta, Christopher R., Xue, Xiaojiao, Mutyam, Venkateshwar, Keeling, Kim M., Mobley, James A., Rowe, Steven M., Bedwell, David M., Welch, Ellen M., Jacobson, Allan

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Ataluren as an Agent for Therapeutic Nonsense Suppression von Peltz, Stuart W, Morsy, Manal, Welch, Ellen M, Jacobson, Allan

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The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokines von Luban, Jeremy, Sattler, Rachel A., Mühlberger, Elke, Graci, Jason D., Cao, Liangxian, Weetall, Marla, Trotta, Christopher, Colacino, Joseph M., Bavari, Sina, Strambio-De-Castillia, Caterina, Suder, Ellen L., Wang, Yetao, Soloveva, Veronica, Cintron-Lue, Katherine, Naryshkin, Nikolai A., Pykett, Mark, Welch, Ellen M., O’Keefe, Kylie, Kong, Ronald, Goodwin, Elizabeth, Jacobson, Allan, Paessler, Slobodan, Peltz, Stuart W.

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Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial von Kerem, Eitan, MD, Konstan, Michael W, Dr, De Boeck, Kris, MD, Accurso, Frank J, MD, Sermet-Gaudelus, Isabelle, MD, Wilschanski, Michael, MD, Elborn, J Stuart, MD, Melotti, Paola, MD, Bronsveld, Inez, MD, Fajac, Isabelle, MD, Malfroot, Anne, MD, Rosenbluth, Daniel B, MD, Walker, Patricia A, MD, McColley, Susanna A, MD, Knoop, Christiane, MD, Quattrucci, Serena, MD, Rietschel, Ernst, MD, Zeitlin, Pamela L, MD, Barth, Jay, MD, Elfring, Gary L, MS, Welch, Ellen M, PhD, Branstrom, Arthur, PhD, Spiegel, Robert J, MD, Peltz, Stuart W, PhD, Ajayi, Temitayo, MD, Rowe, Steven M, MD

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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 minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential von Friesen, Westley J, Johnson, Briana, Sierra, Jairo, Zhuo, Jin, Vazirani, Priya, Xue, Xiaojiao, Tomizawa, Yuki, Baiazitov, Ramil, Morrill, Christie, Ren, Hongyu, Babu, Suresh, Moon, Young-Choon, Branstrom, Art, Mollin, Anna, Hedrick, Jean, Sheedy, Josephine, Elfring, Gary, Weetall, Marla, Colacino, Joseph M, Welch, Ellen M, Peltz, Stuart W

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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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In vitro metabolism, reaction phenotyping, enzyme kinetics, CYP inhibition and induction potential of ataluren von Kong, Ronald, Ma, Jiyuan, Hwang, Seongwoo, Moon, Young‐Choon, Welch, Ellen M., Weetall, Marla, Colacino, Joseph M., Almstead, Neil, Babiak, John, Goodwin, Elizabeth

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Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression von BINGJING WANG, ZHAOHUI YANG, BRISSON, Becky K, HUISHENG FENG, ZHIQIAN ZHANG, WELCH, Ellen M, PELTZ, Stuart W, BARTON, Elisabeth R, BROWN, Robert H, SWEENEY, H. Lee

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Nonsense suppression activity of PTC124 (ataluren) von Peltz, Stuart W, Welch, Ellen M, Jacobson, Allan, Trotta, Christopher R, Naryshkin, Nikolai, Sweeney, H. Lee, Bedwell, David M

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SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy von Naryshkin, Nikolai A., Weetall, Marla, Dakka, Amal, Narasimhan, Jana, Zhao, Xin, Feng, Zhihua, Ling, Karen K. Y., Karp, Gary M., Qi, Hongyan, Woll, Matthew G., Chen, Guangming, Zhang, Nanjing, Gabbeta, Vijayalakshmi, Vazirani, Priya, Bhattacharyya, Anuradha, Furia, Bansri, Risher, Nicole, Sheedy, Josephine, Kong, Ronald, Ma, Jiyuan, Turpoff, Anthony, Lee, Chang-Sun, Zhang, Xiaoyan, Moon, Young-Choon, Trifillis, Panayiota, Welch, Ellen M., Colacino, Joseph M., Babiak, John, Almstead, Neil G., Peltz, Stuart W., Eng, Loren A., Chen, Karen S., Mull, Jesse L., Lynes, Maureen S., Rubin, Lee L., Fontoura, Paulo, Santarelli, Luca, Haehnke, Daniel, McCarthy, Kathleen D., Schmucki, Roland, Ebeling, Martin, Sivaramakrishnan, Manaswini, Ko, Chien-Ping, Paushkin, Sergey V., Ratni, Hasane, Gerlach, Irene, Ghosh, Anirvan, Metzger, Friedrich

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Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder von Voronin, Gregory, Narasimhan, Jana, Gittens, Jamila, Sheedy, Josephine, Lipari, Philip, Peters, Melinda, DeMarco, Steven, Cao, Liangxian, Varganov, Yakov, Kim, Min Jung, Pear, Lisset, Fotouh, Eman, Sinha, Supriya, Ray, Balmiki, Wu, Michael C., Yalamanchili, Padmaja, Southgate, Christopher, Pick, Joseph, Saadipour, Khalil, Jung, Stephen, Lee, Jeanee, Mollin, Anna, Welch, Ellen M., Wu, Zhijian, Weetall, Marla

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

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Guanidino quinazolines and pyrimidines promote readthrough of premature termination codons in cells with native nonsense mutations von Morrill, Christie, Friesen, Westley J., Babu, Suresh, Baiazitov, Ramil Y., Du, Wu, Karloff, Diane B., Lee, Chang-Sun, Moon, Young-Choon, Ren, Hongyu, Sierra, Jairo, Tomizawa, Yuki, Vazirani, Priya, Welch, Ellen M., Xue, Xiaojiao, Zhuo, Jin

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Pharmacologically induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset von Feng, Zhihua, Ling, Karen K Y, Zhao, Xin, Zhou, Chunyi, Karp, Gary, Welch, Ellen M, Naryshkin, Nikolai, Ratni, Hasane, Chen, Karen S, Metzger, Friedrich, Paushkin, Sergey, Weetall, Marla, Ko, Chien-Ping

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The nucleoside analog clitocine is a potent and efficacious readthrough agent von Friesen, Westley J, Trotta, Christopher R, Tomizawa, Yuki, Zhuo, Jin, Johnson, Briana, Sierra, Jairo, Roy, Bijoyita, Weetall, Marla, Hedrick, Jean, Sheedy, Josephine, Takasugi, James, Moon, Young-Choon, Babu, Suresh, Baiazitov, Ramil, Leszyk, John D, Davis, Thomas W, Colacino, Joseph M, Peltz, Stuart W, Welch, Ellen M

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Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy von Zhao, Xin, Feng, Zhihua, Ling, Karen K Y, Mollin, Anna, Sheedy, Josephine, Yeh, Shirley, Petruska, Janet, Narasimhan, Jana, Dakka, Amal, Welch, Ellen M, Karp, Gary, Chen, Karen S, Metzger, Friedrich, Ratni, Hasane, Lotti, Francesco, Tisdale, Sarah, Naryshkin, Nikolai A, Pellizzoni, Livio, Paushkin, Sergey, Ko, Chien-Ping, Weetall, Marla

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Targeting post-transcriptional control for drug discovery von Peltz, Stuart W., Welch, Ellen M., Trotta, Christopher, Davis, Thomas, Jacobson, Allan

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PTC124 for cystic fibrosis – Authors' reply von Kerem, Eitan, Kerem, Batsheva, Welch, Ellen M, Trotta, Christopher, Weetall, Marla, Miller, Langdon, Jacobson, Allan, Peltz, Stuart W

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Corrigendum to: Pharmacologically-induced mouse model of adult spinal muscular atrophy to evaluate effectiveness of therapeutics after disease onset von Feng, Zhihua, Ling, Karen K Y, Zhao, Xin, Zhou, Chunyi, Karp, Gary, Welch, Ellen M, Naryshkin, Nikolai, Ratni, Hasane, Chen, Karen S, Metzger, Friedrich, Paushkin, Sergey, Weetall, Marla, Ko, Chien-Ping

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