Artículos de revistas sobre el tema "Ataluren"
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Michorowska, Sylwia. "Ataluren—Promising Therapeutic Premature Termination Codon Readthrough Frontrunner". Pharmaceuticals 14, n.º 8 (9 de agosto de 2021): 785. http://dx.doi.org/10.3390/ph14080785.
Texto completoDavies, S., N. Serradell, E. Rosa y R. Castañer. "Ataluren". Drugs of the Future 33, n.º 9 (2008): 733. http://dx.doi.org/10.1358/dof.2008.033.09.1252090.
Texto completoMercuri, Eugenio, Francesco Muntoni, Andrés Nascimento Osorio, Már Tulinius, Filippo Buccella, Lauren P. Morgenroth, Heather Gordish-Dressman et al. "Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study". Journal of Comparative Effectiveness Research 9, n.º 5 (abril de 2020): 341–60. http://dx.doi.org/10.2217/cer-2019-0171.
Texto completoKaushik, Diksha, Jiyuan Ma, Guodong Gu, Seongwoo Hwang, Young-Choon Moon y Ronald Kong. "LC–MS/MS quantification of ataluren and ataluren acyl glucuronide in human plasma/urine: application in clinical studies". Bioanalysis 12, n.º 21 (noviembre de 2020): 1545–55. http://dx.doi.org/10.4155/bio-2020-0214.
Texto completoMcDonald, CM, K. Bushby, M. Tulinius, R. Finkel, H. Topaloglu, JW Day, K. Flanigan et al. "A.06 Ataluren: an overview of clinical trial results in nonsense mutation Duchenne Muscular Dystrophy (nmDMD)". Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques 43, S2 (junio de 2016): S8. http://dx.doi.org/10.1017/cjn.2016.58.
Texto completoMcDonald, Craig M., Francesco Muntoni, Vinay Penematsa, Joel Jiang, Allan Kristensen, Francesco Bibbiani, Elizabeth Goodwin et al. "Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients". Journal of Comparative Effectiveness Research 11, n.º 3 (febrero de 2022): 139–55. http://dx.doi.org/10.2217/cer-2021-0196.
Texto completoBeryozkin, Avigail, Ananya Samanta, Prakadeeswari Gopalakrishnan, Samer Khateb, Eyal Banin, Dror Sharon y Kerstin Nagel-Wolfrum. "Translational Read-Through Drugs (TRIDs) Are Able to Restore Protein Expression and Ciliogenesis in Fibroblasts of Patients with Retinitis Pigmentosa Caused by a Premature Termination Codon in FAM161A". International Journal of Molecular Sciences 23, n.º 7 (24 de marzo de 2022): 3541. http://dx.doi.org/10.3390/ijms23073541.
Texto completoRoy, Bijoyita, Westley J. Friesen, Yuki Tomizawa, John D. Leszyk, Jin Zhuo, Briana Johnson, Jumana Dakka et al. "Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression". Proceedings of the National Academy of Sciences 113, n.º 44 (4 de octubre de 2016): 12508–13. http://dx.doi.org/10.1073/pnas.1605336113.
Texto completoDjayet, Celia, Dominique Bremond-Gignac, Justine Touchard, Philippe-Henri Secretan, Fabrice Vidal, Matthieu P. Robert, Alejandra Daruich, Salvatore Cisternino y Joël Schlatter. "Formulation and Stability of Ataluren Eye Drop Oily Solution for Aniridia". Pharmaceutics 13, n.º 1 (22 de diciembre de 2020): 7. http://dx.doi.org/10.3390/pharmaceutics13010007.
Texto completoRyan, Nicola J. "Ataluren: First Global Approval". Drugs 74, n.º 14 (septiembre de 2014): 1709–14. http://dx.doi.org/10.1007/s40265-014-0287-4.
Texto completoCampbell, Craig, Richard J. Barohn, Enrico Bertini, Brigitte Chabrol, Giacomo Pietro Comi, Basil T. Darras, Richard S. Finkel et al. "Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy". Journal of Comparative Effectiveness Research 9, n.º 14 (octubre de 2020): 973–84. http://dx.doi.org/10.2217/cer-2020-0095.
Texto completoNg, Martin Y., Hong Li, Mikel D. Ghelfi, Yale E. Goldman y Barry S. Cooperman. "Ataluren and aminoglycosides stimulate read-through of nonsense codons by orthogonal mechanisms". Proceedings of the National Academy of Sciences 118, n.º 2 (7 de enero de 2021): e2020599118. http://dx.doi.org/10.1073/pnas.2020599118.
Texto completoGhelfi, Mikel D., Saleem Y. Bhat, Hong Li y Barry S. Cooperman. "A High-Throughput Assay for In Vitro Determination of Release Factor-Dependent Peptide Release from a Pretermination Complex by Fluorescence Anisotropy—Application to Nonsense Suppressor Screening and Mechanistic Studies". Biomolecules 13, n.º 2 (27 de enero de 2023): 242. http://dx.doi.org/10.3390/biom13020242.
Texto completoSamanta, Ananya, Katarina Stingl, Susanne Kohl, Jessica Ries, Joshua Linnert y Kerstin Nagel-Wolfrum. "Ataluren for the Treatment of Usher Syndrome 2A Caused by Nonsense Mutations". International Journal of Molecular Sciences 20, n.º 24 (12 de diciembre de 2019): 6274. http://dx.doi.org/10.3390/ijms20246274.
Texto completoGoemans, N., C. Campbell, CM McDonald, T. Voit, X. Luo, G. Elfring, H. Kroger et al. "D.08 ACT DMD (Ataluren Confirmatory Trial in Duchenne Muscular Dystrophy): effect of Ataluren on timed function tests (TFT) in nonsense mutation (nm) DMD". Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques 43, S2 (junio de 2016): S15. http://dx.doi.org/10.1017/cjn.2016.81.
Texto completoBerger, Joachim, Mei Li, Silke Berger, Michelle Meilak, Jeanette Rientjes y Peter D. Currie. "Effect of Ataluren on dystrophin mutations". Journal of Cellular and Molecular Medicine 24, n.º 12 (28 de abril de 2020): 6680–89. http://dx.doi.org/10.1111/jcmm.15319.
Texto completoPeltz, S. W., E. M. Welch, A. Jacobson, C. R. Trotta, N. Naryshkin, H. L. Sweeney y D. M. Bedwell. "Nonsense suppression activity of PTC124 (ataluren)". Proceedings of the National Academy of Sciences 106, n.º 25 (8 de junio de 2009): E64. http://dx.doi.org/10.1073/pnas.0901936106.
Texto completoBezzerri, Valentino, Antonio Vella, Elisabetta D'Aversa, Martina Api, Marisole Allegri, Elena Marinelli Busilacchi, Giovanna D'Amico et al. "Breakthroughs in Preclinical Development of Ataluren (PTC124) As Therapeutic Option for Patients Affected By Shwachman-Diamond Syndrome: Towards the First Clinical Trial". Blood 134, Supplement_1 (13 de noviembre de 2019): 451. http://dx.doi.org/10.1182/blood-2019-127866.
Texto completoPodkletnova, Tatyana V., Lyudmila M. Kuzenkova, Alexey L. Kurenkov, Evgeniya V. Uvakina, Sofya G. Popovich y Anastasiya A. Lyalina. "A clinical case of successful management of a patient with Duchenne muscular dystrophy caused by a nonsense mutation in the DMD gene". L.O. Badalyan Neurological Journal 3, n.º 2 (30 de junio de 2022): 96–100. http://dx.doi.org/10.46563/2686-8997-2022-3-2-96-100.
Texto completoMorkous, Sameh S. "Treatment with Ataluren for Duchene Muscular Dystrophy". Pediatric Neurology Briefs 34 (4 de diciembre de 2020): 12. http://dx.doi.org/10.15844/pedneurbriefs-34-12.
Texto completoShoseyov, David, Malena Cohen-Cymberknoh y Michael Wilschanski. "Ataluren for the treatment of cystic fibrosis". Expert Review of Respiratory Medicine 10, n.º 4 (24 de febrero de 2016): 387–91. http://dx.doi.org/10.1586/17476348.2016.1150181.
Texto completoSiddiqui, Nadeem y Nahum Sonenberg. "Proposing a mechanism of action for ataluren". Proceedings of the National Academy of Sciences 113, n.º 44 (19 de octubre de 2016): 12353–55. http://dx.doi.org/10.1073/pnas.1615548113.
Texto completoMa, Jiyuan, Nicole Risher, Valerie Northcutt, Young-Choon Moon, Marla Weetall, Ellen Welch, Joseph Colacino, Neil Almstead y Ronald Kong. "Ataluren metabolism: Ataluren-O-1β-acyl glucuronide is a stable circulating metabolite in mouse, rat, dog and human". Drug Metabolism and Pharmacokinetics 38 (junio de 2021): 100393. http://dx.doi.org/10.1016/j.dmpk.2021.100393.
Texto completoMuntoni, Francesco, Isabelle Desguerre, Michela Guglieri, Andrés Nascimento Osorio, Janbernd Kirschner, Már Tulinius, Filippo Buccella et al. "Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry". Journal of Comparative Effectiveness Research 8, n.º 14 (octubre de 2019): 1187–200. http://dx.doi.org/10.2217/cer-2019-0086.
Texto completoBezzerri, Valentino, Laura Lentini, Martina Api, Elena Marinelli Busilacchi, Vincenzo Cavalieri, Antonella Pomilio, Francesca Diomede et al. "Novel Translational Read-through–Inducing Drugs as a Therapeutic Option for Shwachman-Diamond Syndrome". Biomedicines 10, n.º 4 (12 de abril de 2022): 886. http://dx.doi.org/10.3390/biomedicines10040886.
Texto completoMercuri, Eugenio, Ros Quinlivan y Sylvie Tuffery-Giraud. "Early Diagnosis and Treatment – The Use of Ataluren in the Effective Management of Duchenne Muscular Dystrophy". European Neurological Review 13, n.º 1 (2018): 31. http://dx.doi.org/10.17925/enr.2018.13.1.31.
Texto completoWang, Bingjing, Zhaohui Yang, Becky K. Brisson, Huisheng Feng, Zhiqian Zhang, Ellen M. Welch, Stuart W. Peltz, Elisabeth R. Barton, Robert H. Brown y H. Lee Sweeney. "Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppression". Journal of Applied Physiology 109, n.º 3 (septiembre de 2010): 901–5. http://dx.doi.org/10.1152/japplphysiol.01366.2009.
Texto completo&NA;. "Ataluren a no nonsense approach to cystic fibrosis". Inpharma Weekly &NA;, n.º 1653 (agosto de 2008): 7. http://dx.doi.org/10.2165/00128413-200816530-00011.
Texto completoPeltz, Stuart W., Manal Morsy, Ellen M. Welch y Allan Jacobson. "Ataluren as an Agent for Therapeutic Nonsense Suppression". Annual Review of Medicine 64, n.º 1 (14 de enero de 2013): 407–25. http://dx.doi.org/10.1146/annurev-med-120611-144851.
Texto completoLoudon, John A. "Ataluren: A ‘no-nonsense’ approach for pulmonary diseases". Pulmonary Pharmacology & Therapeutics 26, n.º 3 (junio de 2013): 398–99. http://dx.doi.org/10.1016/j.pupt.2013.01.007.
Texto completoBushby, Katharine, Richard Finkel, Brenda Wong, Richard Barohn, Craig Campbell, Giacomo P. Comi, Anne M. Connolly et al. "Ataluren treatment of patients with nonsense mutation dystrophinopathy". Muscle & Nerve 50, n.º 4 (22 de septiembre de 2014): 477–87. http://dx.doi.org/10.1002/mus.24332.
Texto completoKong, Ronald, Oscar L. Laskin, Diksha Kaushik, Fengbin Jin, Jiyuan Ma, Joseph McIntosh, Marcio Souza y Neil Almstead. "Ataluren Pharmacokinetics in Healthy Japanese and Caucasian Subjects". Clinical Pharmacology in Drug Development 8, n.º 2 (10 de enero de 2019): 172–78. http://dx.doi.org/10.1002/cpdd.645.
Texto completoKent, Emma y Thomas Paling. "VP16 A NICE Way To Manage Managed Access: Case Study In Muscular Dystrophy". International Journal of Technology Assessment in Health Care 35, S1 (2019): 79. http://dx.doi.org/10.1017/s0266462319002939.
Texto completoDrake, Kylie M., Benjamin J. Dunmore, Lauren N. McNelly, Nicholas W. Morrell y Micheala A. Aldred. "Correction of NonsenseBMPR2andSMAD9Mutations by Ataluren in Pulmonary Arterial Hypertension". American Journal of Respiratory Cell and Molecular Biology 49, n.º 3 (septiembre de 2013): 403–9. http://dx.doi.org/10.1165/rcmb.2013-0100oc.
Texto completoWilschanski, M., L. L. Miller, D. Shoseyov, H. Blau, J. Rivlin, M. Aviram, M. Cohen et al. "Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis". European Respiratory Journal 38, n.º 1 (13 de enero de 2011): 59–69. http://dx.doi.org/10.1183/09031936.00120910.
Texto completoHall, Adam, Lok Wan Liu, Richard Macaulay y Sean Walsh. "OP127 Sugar And Spice And All Things NICE - Managed Access Agreements". International Journal of Technology Assessment in Health Care 35, S1 (2019): 28–29. http://dx.doi.org/10.1017/s0266462319001582.
Texto completoLoudon, John A. "Ataluren-time for a 'no–nonsense' approach to haematological malignancies". Hematology and Leukemia 1, n.º 1 (2013): 2. http://dx.doi.org/10.7243/2052-434x-1-2.
Texto completoLoudon, John A. "Ataluren: Time for a ‘No-Nonsense’ Approach to Heart Diseases". Cardiovascular Drugs and Therapy 27, n.º 2 (6 de enero de 2013): 181–82. http://dx.doi.org/10.1007/s10557-012-6435-8.
Texto completoBlaschek, Astrid, Martin Rodrigues, Lena Ille, Mohammed Idriess, Therese Well, Birgit Warken, Christine Müller et al. "Is Exercise-Induced Fatigue a Problem in Children with Duchenne Muscular Dystrophy?" Neuropediatrics 51, n.º 05 (5 de mayo de 2020): 342–48. http://dx.doi.org/10.1055/s-0040-1708859.
Texto completoWang, Xia, Xianghong Shan, Kevin Gregory-Evans y Cheryl Y. Gregory-Evans. "RNA-based therapies in animal models of Leber congenital amaurosis causing blindness". Precision Clinical Medicine 3, n.º 2 (12 de marzo de 2020): 113–26. http://dx.doi.org/10.1093/pcmedi/pbaa009.
Texto completoWerner, C., X. Luo, G. Elfring, H. Kroger, P. Riebling, T. Ong, R. Spiegel y S. Peltz. "Meta-analyses of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy". Neuropediatrics 48, S 01 (26 de abril de 2017): S1—S45. http://dx.doi.org/10.1055/s-0037-1602954.
Texto completoMuntoni, F., E. Mercuri, X. Luo, G. Elfring, C. Werner, P. Trifillis, S. W. Peltz y C. M. McDonald. "Meta-analyses of ataluren in patients with nonsense mutation Duchenne muscular dystrophy". Neuromuscular Disorders 28 (abril de 2018): S12. http://dx.doi.org/10.1016/s0960-8966(18)30323-7.
Texto completoZainal Abidin, Noreen, Iram J. Haq, Aaron I. Gardner y Malcolm Brodlie. "Ataluren in cystic fibrosis: development, clinical studies and where are we now?" Expert Opinion on Pharmacotherapy 18, n.º 13 (1 de agosto de 2017): 1363–71. http://dx.doi.org/10.1080/14656566.2017.1359255.
Texto completoSeo, K., D. Kim, H. Lee y J. Shin. "FP.33 Ataluren treatment in 30-week-old dysferlinopathy mouse with nonsense mutation". Neuromuscular Disorders 32 (octubre de 2022): S112. http://dx.doi.org/10.1016/j.nmd.2022.07.293.
Texto completoBhattacharya, Arpan, Shijie Huang, Mikel D. Ghelfi, Hong Li, Clark Fritsch, David M. Chenoweth, Barry S. Cooperman y Yale E. Goldman. "Elucidating the mechanism and target sites of ataluren inside the protein synthesis machinery". Biophysical Journal 121, n.º 3 (febrero de 2022): 203a. http://dx.doi.org/10.1016/j.bpj.2021.11.1723.
Texto completoReeves, Emer P., Ciara A. O’Dwyer, Danielle M. Dunlea, Mark R. Wormald, Padraig Hawkins, Mohammad Alfares, Darrell N. Kotton, Steven M. Rowe, Andrew A. Wilson y Noel G. McElvaney. "Ataluren, a New Therapeutic for Alpha-1 Antitrypsin–Deficient Individuals with Nonsense Mutations". American Journal of Respiratory and Critical Care Medicine 198, n.º 8 (15 de octubre de 2018): 1099–102. http://dx.doi.org/10.1164/rccm.201802-0338le.
Texto completoWalsh, Sean, Ricky Tsang, Erika Turkstra y Richard Macaulay. "VP22 Future Trends For Managed Access Agreements In The UK". International Journal of Technology Assessment in Health Care 34, S1 (2018): 164–65. http://dx.doi.org/10.1017/s0266462318003446.
Texto completoMartínez-Edo, Gabriel, Maria C. Llinàs, Salvador Borrós y David Sánchez-García. "Isothiocyanate-Functionalized Mesoporous Silica Nanoparticles as Building Blocks for the Design of Nanovehicles with Optimized Drug Release Profile". Nanomaterials 9, n.º 9 (29 de agosto de 2019): 1219. http://dx.doi.org/10.3390/nano9091219.
Texto completoTutone, Marco, Ivana Pibiri, Laura Lentini, Andrea Pace y Anna Maria Almerico. "Deciphering the Nonsense Readthrough Mechanism of Action of Ataluren: An in Silico Compared Study". ACS Medicinal Chemistry Letters 10, n.º 4 (7 de febrero de 2019): 522–27. http://dx.doi.org/10.1021/acsmedchemlett.8b00558.
Texto completoDe Boeck, K., H. G. M. Heijerman, J. C. Davies, I. Sermet-Gaudelus, L. Hjelte, E. Kerem, J. Sun, J. Mcintosh, A. Malfroot y H. A. W. M. Tiddens. "WS13.1 Ataluren significantly reduces exacerbations in nonsense mutation cystic fibrosis patients not receiving tobramycin". Journal of Cystic Fibrosis 15 (junio de 2016): S20—S21. http://dx.doi.org/10.1016/s1569-1993(16)30132-1.
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