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ArticlesPeer ReviewedFull Text
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Author / Creator:Mercuri, Eugenio ; Darras, Basil T ; Chiriboga, Claudia A ; Day, John W ; Campbell, Craig ; Connolly, Anne M ; Iannaccone, Susan T ; Kirschner, Janbernd ; Kuntz, Nancy L ; Saito, Kayoko ; Shieh, Perry B ; Tulinius, Már ; Mazzone, Elena S ; Montes, Jacqueline ; Bishop, Kathie M ; Yang, Qingqing ; Foster, Richard ; Gheuens, Sarah ; Bennett, C. Frank ; Farwell, Wildon ; Schneider, Eugene ; De Vivo, Darryl C ; Finkel, Richard S
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2018-02, Vol.378 (7), p.625-635
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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
Author / Creator:Finkel, Richard S, Dr ; Chiriboga, Claudia A, MD ; Vajsar, Jiri, MD ; Day, John W, MD ; Montes, Jacqueline, EdD ; De Vivo, Darryl C, MD ; Yamashita, Mason, MD ; Rigo, Frank, PhD ; Hung, Gene, MD ; Schneider, Eugene, MD ; Norris, Daniel A, PhD ; Xia, Shuting, MS ; Bennett, C Frank, PhD ; Bishop, Kathie M, PhD
Publisher:England: Elsevier Ltd
Journal title:The Lancet (British edition), 2016-12, Vol.388 (10063), p.3017-3026
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Lipoprotein(a) Reduction in Persons with Cardiovascular Disease
Author / Creator:Tsimikas, Sotirios ; Karwatowska-Prokopczuk, Ewa ; Gouni-Berthold, Ioanna ; Tardif, Jean-Claude ; Baum, Seth J ; Steinhagen-Thiessen, Elizabeth ; Shapiro, Michael D ; Stroes, Erik S ; Moriarty, Patrick M ; Nordestgaard, Børge G ; Xia, Shuting ; Guerriero, Jonathan ; Viney, Nicholas J ; O’Dea, Louis ; Witztum, Joseph L
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2020-01, Vol.382 (3), p.244-255
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Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
Author / Creator:Miller, Timothy ; Cudkowicz, Merit ; Shaw, Pamela J ; Andersen, Peter M ; Atassi, Nazem ; Bucelli, Robert C ; Genge, Angela ; Glass, Jonathan ; Ladha, Shafeeq ; Ludolph, Albert L ; Maragakis, Nicholas J ; McDermott, Christopher J ; Pestronk, Alan ; Ravits, John ; Salachas, François ; Trudell, Randall ; Van Damme, Philip ; Zinman, Lorne ; Bennett, C. Frank ; Lane, Roger ; Sandrock, Alfred ; Runz, Heiko ; Graham, Danielle ; Houshyar, Hani ; McCampbell, Alexander ; Nestorov, Ivan ; Chang, Ih ; McNeill, Manjit ; Fanning, Laura ; Fradette, Stephanie ; Ferguson, Toby A
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2020-07, Vol.383 (2), p.109-119
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ArticlesPeer ReviewedFull Text
Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome
Author / Creator:Witztum, Joseph L ; Gaudet, Daniel ; Freedman, Steven D ; Alexander, Veronica J ; Digenio, Andres ; Williams, Karren R ; Yang, Qingqing ; Hughes, Steven G ; Geary, Richard S ; Arca, Marcello ; Stroes, Erik S.G ; Bergeron, Jean ; Soran, Handrean ; Civeira, Fernando ; Hemphill, Linda ; Tsimikas, Sotirios ; Blom, Dirk J ; O’Dea, Louis ; Bruckert, Eric
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2019-08, Vol.381 (6), p.531-542
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ArticlesPeer ReviewedFull Text
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Author / Creator:Finkel, Richard S ; Mercuri, Eugenio ; Darras, Basil T ; Connolly, Anne M ; Kuntz, Nancy L ; Kirschner, Janbernd ; Chiriboga, Claudia A ; Saito, Kayoko ; Servais, Laurent ; Tizzano, Eduardo ; Topaloglu, Haluk ; Tulinius, Már ; Montes, Jacqueline ; Glanzman, Allan M ; Bishop, Kathie ; Zhong, Z. John ; Gheuens, Sarah ; Bennett, C. Frank ; Schneider, Eugene ; Farwell, Wildon ; De Vivo, Darryl C
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2017-11, Vol.377 (18), p.1723-1732
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ArticlesPeer ReviewedFull Text
Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia
Author / Creator:Gaudet, Daniel ; Alexander, Veronica J ; Baker, Brenda F ; Brisson, Diane ; Tremblay, Karine ; Singleton, Walter ; Geary, Richard S ; Hughes, Steven G ; Viney, Nicholas J ; Graham, Mark J ; Crooke, Rosanne M ; Witztum, Joseph L ; Brunzell, John D ; Kastelein, John J.P
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2015-07, Vol.373 (5), p.438-447
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ArticlesPeer ReviewedFull Text
Factor XI Antisense Oligonucleotide for Prevention of Venous Thrombosis
Author / Creator:Büller, Harry R ; Bethune, Claudette ; Bhanot, Sanjay ; Gailani, David ; Monia, Brett P ; Raskob, Gary E ; Segers, Annelise ; Verhamme, Peter ; Weitz, Jeffrey I
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2015-01, Vol.372 (3), p.232-240
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ArticlesPeer ReviewedFull Text
Treatment of HCV Infection by Targeting MicroRNA
Author / Creator:Janssen, Harry L.A ; Reesink, Hendrik W ; Lawitz, Eric J ; Zeuzem, Stefan ; Rodriguez-Torres, Maribel ; Patel, Keyur ; van der Meer, Adriaan J ; Patick, Amy K ; Chen, Alice ; Zhou, Yi ; Persson, Robert ; King, Barney D ; Kauppinen, Sakari ; Levin, Arthur A ; Hodges, Michael R
Publisher:Waltham, MA: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2013-05, Vol.368 (18), p.1685-1694
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ArticlesPeer ReviewedFull Text
Mongersen, an Oral SMAD7 Antisense Oligonucleotide, and Crohn’s Disease
Author / Creator:Monteleone, Giovanni ; Neurath, Markus F ; Ardizzone, Sandro ; Di Sabatino, Antonio ; Fantini, Massimo C ; Castiglione, Fabiana ; Scribano, Maria L ; Armuzzi, Alessandro ; Caprioli, Flavio ; Sturniolo, Giacomo C ; Rogai, Francesca ; Vecchi, Maurizio ; Atreya, Raja ; Bossa, Fabrizio ; Onali, Sara ; Fichera, Maria ; Corazza, Gino R ; Biancone, Livia ; Savarino, Vincenzo ; Pica, Roberta ; Orlando, Ambrogio ; Pallone, Francesco
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2015-03, Vol.372 (12), p.1104-1113
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ArticlesPeer ReviewedFull Text
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study
Author / Creator:Voit, Thomas, Prof ; Topaloglu, Haluk, Prof ; Straub, Volker, Prof ; Muntoni, Francesco, Prof ; Deconinck, Nicolas, MD ; Campion, Giles, MD ; De Kimpe, Sjef J, PhD ; Eagle, Michelle, PhD ; Guglieri, Michela, MD ; Hood, Steve, PhD ; Liefaard, Lia, PhD ; Lourbakos, Afrodite, PhD ; Morgan, Allison, BSc ; Nakielny, Joanna, MB ; Quarcoo, Naashika, PhD ; Ricotti, Valeria, MB ; Rolfe, Katie, MSc ; Servais, Laurent, MD ; Wardell, Claire, DPhil ; Wilson, Rosamund, PhD ; Wright, Padraig, MD ; Kraus, John E, MD
Publisher:England: Elsevier Ltd
Journal title:Lancet neurology, 2014-10, Vol.13 (10), p.987-996
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ArticlesPeer ReviewedFull Text
Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome
Author / Creator:Stroes, Erik S.G. ; Alexander, Veronica J. ; Karwatowska-Prokopczuk, Ewa ; Hegele, Robert A. ; Arca, Marcello ; Ballantyne, Christie M. ; Soran, Handrean ; Prohaska, Thomas A. ; Xia, Shuting ; Ginsberg, Henry N. ; Witztum, Joseph L. ; Tsimikas, Sotirios
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2024-05, Vol.390 (19), p.1781-1792
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Olezarsen for Hypertriglyceridemia in Patients at High Cardiovascular Risk
Author / Creator:Bergmark, Brian A. ; Marston, Nicholas A. ; Prohaska, Thomas A. ; Alexander, Veronica J. ; Zimerman, André ; Moura, Filipe A. ; Murphy, Sabina A. ; Goodrich, Erica L. ; Zhang, Shuanglu ; Gaudet, Daniel ; Karwatowska-Prokopczuk, Ewa ; Tsimikas, Sotirios ; Giugliano, Robert P. ; Sabatine, Marc S.
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2024-05, Vol.390 (19), p.1770-1780
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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
Author / Creator:Kinali, Maria, MD ; Arechavala-Gomeza, Virginia, PhD ; Feng, Lucy, PhD ; Cirak, Sebahattin, MD ; Hunt, David, FRCS ; Adkin, Carl, PhD ; Guglieri, Michela, MD ; Ashton, Emma, PhD ; Abbs, Stephen, PhD ; Nihoyannopoulos, Petros, MD ; Garralda, Maria Elena, MD ; Rutherford, Mary, MD ; Mcculley, Caroline, PhD ; Popplewell, Linda, PhD ; Graham, Ian R, PhD ; Dickson, George, PhD ; Wood, Matthew JA, MD ; Wells, Dominic J, PhD ; Wilton, Steve D, PhD ; Kole, Ryszard, PhD ; Straub, Volker, MD ; Bushby, Kate, MD ; Sewry, Caroline, PhD ; Morgan, Jennifer E, PhD ; Muntoni, Francesco, MD
Publisher:England: Elsevier Ltd
Journal title:Lancet neurology, 2009-10, Vol.8 (10), p.918-928
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ArticlesPeer ReviewedFull Text
LNA-mediated microRNA silencing in non-human primates
Author / Creator:Elmén, Joacim ; Lindow, Morten ; Schütz, Sylvia ; Lawrence, Matthew ; Petri, Andreas ; Obad, Susanna ; Lindholm, Marie ; Hedtjärn, Maj ; Hansen, Henrik Frydenlund ; Berger, Urs ; Gullans, Steven ; Kearney, Phil ; Sarnow, Peter ; Straarup, Ellen Marie ; Kauppinen, Sakari
Publisher:London: Nature Publishing Group UK
Journal title:Nature, 2008-04, Vol.452 (7189), p.896-899
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Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection
Author / Creator:Lanford, Robert E ; Hildebrandt-Eriksen, Elisabeth S ; Petri, Andreas ; Persson, Robert ; Lindow, Morten ; Munk, Martin E ; Kauppinen, Sakari ; Ørum, Henrik
Publisher:Washington, DC: American Association for the Advancement of Science
Journal title:Science (American Association for the Advancement of Science), 2010-01, Vol.327 (5962), p.198-201
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ArticlesPeer ReviewedFull Text
Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy
Author / Creator:Goemans, Nathalie M ; Tulinius, Mar ; van den Akker, Johanna T ; Burm, Brigitte E ; Ekhart, Peter F ; Heuvelmans, Niki ; Holling, Tjadine ; Janson, Anneke A ; Platenburg, Gerard J ; Sipkens, Jessica A ; Sitsen, J.M. Ad ; Aartsma-Rus, Annemieke ; van Ommen, Gert-Jan B ; Buyse, Gunnar ; Darin, Niklas ; Verschuuren, Jan J ; Campion, Giles V ; de Kimpe, Sjef J ; van Deutekom, Judith C
Publisher:Waltham, MA: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2011-04, Vol.364 (16), p.1513-1522
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A Phase-Appropriate Risk Assessment Strategy in Support of the Safety of Peptide and Oligonucleotide-Related Impurities
Author / Creator:Pack, Brian W. ; Siegel, Robert W. ; Cornwell, Paul D. ; Ferrante, Andrea ; Roepke, Douglas A. ; Hodsdon, Michael E. ; Malherbe, Laurent ; Carfagna, Mark A.
Publisher:Cham: Springer International Publishing
Journal title:The AAPS journal, 2025-03, Vol.27 (2), p.56
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Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-cont...
Author / Creator:Raal, Frederick J, Prof ; Santos, Raul D, MD ; Blom, Dirk J, MD ; Marais, A David, Prof ; Charng, Min-Ji, MD ; Cromwell, William C, MD ; Lachmann, Robin H, MRCP ; Gaudet, Daniel, MD ; Tan, Ju L, MB BS ; Chasan-Taber, Scott, PhD ; Tribble, Diane L, PhD ; Flaim, JoAnn D, PhD ; Crooke, Stanley T, MD
Publisher:Kidlington: Elsevier Ltd
Journal title:The Lancet (British edition), 2010-03, Vol.375 (9719), p.998-1006
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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051
Author / Creator:van Deutekom, Judith C ; Janson, Anneke A ; Ginjaar, Ieke B ; Frankhuizen, Wendy S ; Aartsma-Rus, Annemieke ; Bremmer-Bout, Mattie ; den Dunnen, Johan T ; Koop, Klaas ; van der Kooi, Anneke J ; Goemans, Nathalie M ; de Kimpe, Sjef J ; Ekhart, Peter F ; Venneker, Edna H ; Platenburg, Gerard J ; Verschuuren, Jan J ; van Ommen, Gert-Jan B
Publisher:United States: Massachusetts Medical Society
Journal title:The New England journal of medicine, 2007-12, Vol.357 (26), p.2677-2686
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Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

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Lipoprotein(a) Reduction in Persons with Cardiovascular Disease

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Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS

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Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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Antisense Inhibition of Apolipoprotein C-III in Patients with Hypertriglyceridemia

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Factor XI Antisense Oligonucleotide for Prevention of Venous Thrombosis

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Treatment of HCV Infection by Targeting MicroRNA

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Mongersen, an Oral SMAD7 Antisense Oligonucleotide, and Crohn’s Disease

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Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study

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Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome

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Olezarsen for Hypertriglyceridemia in Patients at High Cardiovascular Risk

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Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

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LNA-mediated microRNA silencing in non-human primates

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Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection

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Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

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A Phase-Appropriate Risk Assessment Strategy in Support of the Safety of Peptide and Oligonucleotide-Related Impurities

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Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-cont...

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Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

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