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Nusinersen versus sham control in infantile-onset spinal muscular atrophy

Title:	Nusinersen versus sham control in infantile-onset spinal muscular atrophy
Authors:	Finkel R. S.; Mercuri E.; Darras B. T.; Connolly A. M.; Kuntz N. L.; Kirschner J.; Chiriboga C. A.; Saito K.; Servais L.; Tizzano E.; Topaloglu H.; Tulinius M.; Montes J.; Glanzman A. M.; Bishop K.; Zhong Z. J.; Gheuens S.; Bennett C. F.; Schneider E.; Farwell W.; De Vivo D. C.; Bradley W. G.; Schroth M. K.; Bodensteriner J. B.; Davis C. S.; Shell R.; Hen J.; Austin E. D.; Aziz-Zaman S.; Cappell J.; Constantinescu A.; Cruz R.; Dastgir J.; Dunaway S.; Engelstad K.; Gormley M.; Holuba La Marca N.; Khandji A.; Kramer S.; Marra J.; Ortiz-Miller C.; Popolizio M.; Salazar R.; Sanabria L.; Weimer L.; Anand P.; Gadeken R.; Golumbek P. T.; Siener C.; Zaidman C. M.; Al-Ghamdi F.; Berde C.; Ghosh P.; Graham R.; Harrington T.; Koka A.; Laine R.; Liew W.; Mirek E.; Ordonez G.; Pasternak A.; Quigley J.; Sethna N.; Souris M.; Szelag H.; Wand L.; Day J. W.; D'Souza G.; Duong T. T.; Gee R.; Kitsuwa-Lowe J.; McFall D.; Patnaik S.; Paulose S.; Perez J.; Proud C.; Purse B.; Ramamurthi R. J.; Sakamuri S.; Sampson J.; Sanjanwala B.; Tesi Rocha A. C.; Watson K.; Welsh L.; Pena L. D. M.; Case L.; Coates J.; DeArmey S.; Homi M. M.; Milleson C.; Nelson N.; Ross A.; Smith E.; Taicher B.; Wootton J.; Finanger E.; Benjamin D.; Frank A.; Roberts C.; Russman B.; Zilke K.; Berry D.; Civitello M.; Cook D.; Endsley J. D.; Johnson C.; Kasper M.; Leon W.; Lim A.; O'Reardon K.; Sigurdardottir L. Y.; Turner J.; Weber-Guzman F.; Zinn M.; Iannaccone S. T.; Castro D.; Cowie M.; Farrow-Gillespie A.; Herbert A.; Kauk M.; McElroy D.; Miller N.; Nelson L.; Smith L.; Spain T.; Trest S.; Johnson N.; Butterfield R.; DiBella D.; Mayne K.; Newcomb T. M.; Rausch N.; Blomgren C.; Choi H. W.; Epstein L.; Goldman S.; Krosschell K.; Krueger J.; Kurz J.; Rao V.; Parsons J.; Allen V.; Bielsky A.; Booker K.; Camuto A.; Carry T.; Fuhr P.; Gibbons M.; Janas J.; Johnson H.; Kelly C.; Lord-Halvorson L. S.; Nicolarsen S.; Shea S.; Tran V.; VanderVeen G.; Yang M.; Zimmerman C.; Shieh P.; Parziale N.; Rao L.; Said J. W.; Shu F.; Skura C.; Staudt L.; Tennekoon G.; Adang L.; Brandsema J.; Chadehumbe M.; Flickinger J.; Kichula E.; Stanford D.; Toms M.; Zigmont J.; Oskoui M.; Arpin S.; Dinunzio P.; Ingelmo P. M.; Poulin C.; Rivera G.; Sabapathy C.; Srour M.; Turgeon-Desilet S.; Zielinski D.; Selby K.; King C.; Lee J.; Michoulas A.; Roland E.; Vajsar J.; Chau V.; Dowling J.; Haldenby R.; Miki M.; So S.; Pascual Pascual S. I.; Martinez Bermejo A.; Epinosa Garcia S.; Garcia Guixot S.; Martinez Moreno M. M.; del Pilar Tirado Requero M.; del Mar Garcia Romero M.; Aguilar C.; Munell Casadesus F.; Gomez Garcia de la Banda M. B.; Gallardo M.; Gili G.; Alavarez Molinero M.; de Los Angeles Tormos Munoz M.; Palacios N. J.; Planas Pascual B.; del Mar Melendez Plumed M.; Rucian A. F.; Toro Tamargo E.; Gratacos Vinola M.; Borell S.; Eckenweiler M.; Kruger M.; Pechmann A.; Rippberger B.; Stein S.; Vogt S.; Wider S.; Schara U.; Andres B.; Della Marina A.; Ganfuss A.; Jachertz P.; Koelbel H.; Rupprich K.; Schroers E. S.; Sponemann N.; Bruno C.; Fiorillo C.; Garaventa A.; Lanteri P.; Lanzillotta V.; Manzitti C.; Pedermonte M.; Tacchetti P.; Trucco F.; Zuffi A.; De Sanctis R.; Fanelli L.; Luigetti M.; Palermo C.; Pane M.; Piastra M.; Sivo S.; Gargaun E.; Gidaro T.; Gilabert S.; Leger P. L.; Le Moing A. G.; Lilien C.; Mayer M.; Ollievier Q.; Rambaud J.; Taytard J.; Vialle R.; Voit T.; Muntoni F.; D'Argenzio L.; Lister PH.; Manzur A.; Pisco Domingos J.; Ramsey D.; Ricotti V.; Schottlaender L.; Scoto M.; Scuplak S. M.; Selby V.; Straub V.; Baily S.; Bertoli M.; Mayhew A. G.; Lofra R. M.; Murphy A.; Wood C.; Darin N.; Eldblom J.; Kimber E.; Kroksmark A. K.; Lindstedt A.; Michael E.; Sofou K.; Deconinck N.; Christiaens A.; Coppens S.; DeCock K.; De Vos voor E.; Dorban F.; Gilbert G.; Rooze S.; Tahon V.; Van Coster R.; Van Der Looven R.; Vanlander A.; Vens D.; Verhelst H.; Wenderickx B.; Wittevrongel S.; Farrar M.; Berthon-Jones N.; Doumit M. A.; Herbert K. J.; Kandula T.; Morrison M.; O'Brien J.; Richardson S.; Ferreira Sampaio H. A.; Teoh H. L.; Ryan M.; Carroll K. M.; De Valle K. L.; Villano D.; Woodcock I.; Yiu E. M.; Ardicli D.; Gunbey C.; Haliloglu V. G.; Karaduman A. A.; Konuskan B.; Yildiz Sarikaya F. G.; Serdaroglu E.; Tanyildiz M.; Temucin C. M.; Yildirim M.; Yilmaz O. T.; Arakawa R.; Chiba Y.; Eto K.; Hirasawa K.; Ikai T.; Ito S.; Ito Y.; Kaburagi Y.; Kaneko H.; Matsumaru S.; Matsushima N.; Mizuochi K.; Nagata S.; Nakatsukasa H.; Nishikawa A.; Otani Y.; Sato T.; Shichiji M.; Sugimoto K.; Takeshita A.; Yanagishita T.; Yamauchi A.; Takeshima Y.; Fujino T.; Fukuda N.; Lee T.; Oriyama K.; Shibano T.; Shimomura H.; Tachikawa T.; Tanaka Y.; Taniguchi N.; Chae J. H.; Choi S. A.; Chun S. M.; Jo H.; Kim H.; Kim S. Y.; Lee J. S.; Lim B. C.; Shin H. I.; Son W. S.; Chan S.; Chung A. C.; Yan C. S.; Stella C.; Joseph C. K. W.; Ng C. S.; Alvin H. C. C.; Janice I. J. K.; Wendy L. W. M.; Chui-San M. N.; Ki N. Y.; Shun T. N.; Connie W. Y.; Virginia W. C.; Yvonne Y.; Jong Y. J.; Chen T. H.; Chou P. C.; Chou Y. H.; Chung H. W.; Hsu J. H.; Ju Y. H.; Liang W. C.; Shih H. H.; Wang H. Y.; Wu Y. C.; Zeng Y. S.
Contributors:	Finkel, R; Mercuri, E; Darras, B; Connolly, A; Kuntz, N; Kirschner, J; Chiriboga, C; Saito, K; Servais, L; Tizzano, E; Topaloglu, H; Tulinius, M; Montes, J; Glanzman, A; Bishop, K; Zhong, Z; Gheuens, S; Bennett, C; Schneider, E; Farwell, W; De Vivo, D; Bradley, W; Schroth, M; Bodensteriner, J; Davis, C; Shell, R; Hen, J; Austin, E; Aziz-Zaman, S; Cappell, J; Constantinescu, A; Cruz, R; Dastgir, J; Dunaway, S; Engelstad, K; Gormley, M; Holuba La Marca, N; Khandji, A; Kramer, S; Marra, J; Ortiz-Miller, C; Popolizio, M; Salazar, R; Sanabria, L; Weimer, L; Anand, P; Gadeken, R; Golumbek, P; Siener, C; Zaidman, C; Al-Ghamdi, F; Berde, C; Ghosh, P; Graham, R; Harrington, T; Koka, A; Laine, R; Liew, W; Mirek, E; Ordonez, G; Pasternak, A; Quigley, J; Sethna, N; Souris, M; Szelag, H; Wand, L; Day, J; D'Souza, G; Duong, T; Gee, R; Kitsuwa-Lowe, J; Mcfall, D; Patnaik, S; Paulose, S; Perez, J; Proud, C; Purse, B; Ramamurthi, R; Sakamuri, S; Sampson, J; Sanjanwala, B; Tesi Rocha, A; Watson, K; Welsh, L; Pena, L; Case, L; Coates, J; Dearmey, S; Homi, M; Milleson, C; Nelson, N; Ross, A; Smith, E; Taicher, B; Wootton, J; Finanger, E; Benjamin, D; Frank, A; Roberts, C; Russman, B
Publisher Information:	Massachussetts Medical Society; US
Publication Year:	2017
Collection:	Università degli Studi di Milano-Bicocca: BOA (Bicocca Open Archive)
Subject Terms:	Age of Onset; Disease-Free Survival; Double-Blind Method; Female; Human; Infant; Injections; Spinal; Male; Motor Skill; Oligonucleotide; Oligonucleotides; Antisense; Respiration; Artificial; RNA; Messenger; Spinal Muscular Atrophies of Childhood; Survival Analysi; Survival of Motor Neuron 2 Protein
Description:	BACKGROUND: Spinal muscular atrophy is an autosomal recessive neuromuscular disorder that is caused by an insufficient level of survival motor neuron (SMN) protein. Nusinersen is an antisense oligonucleotide drug that modifies pre–messenger RNA splicing of the SMN2 gene and thus promotes increased production of full-length SMN protein. METHODS: We conducted a randomized, double-blind, sham-controlled, phase 3 efficacy and safety trial of nusinersen in infants with spinal muscular atrophy. The primary end points were a motor-milestone response (defined according to results on the Hammersmith Infant Neurological Examination) and event-free survival (time to death or the use of permanent assisted ventilation). Secondary end points included overall survival and subgroup analyses of event-free survival according to disease duration at screening. Only the first primary end point was tested in a prespecified interim analysis. To control the overall type I error rate at 0.05, a hierarchical testing strategy was used for the second primary end point and the secondary end points in the final analysis. RESULTS: In the interim analysis, a significantly higher percentage of infants in the nusinersen group than in the control group had a motor-milestone response (21 of 51 infants [41%] vs. 0 of 27 [0%], P
Document Type:	article in journal/newspaper
File Description:	STAMPA
Language:	English
Relation:	info:eu-repo/semantics/altIdentifier/pmid/29091570; info:eu-repo/semantics/altIdentifier/wos/WOS:000414239100005; volume:377; issue:18; firstpage:1723; lastpage:1732; numberofpages:10; journal:THE NEW ENGLAND JOURNAL OF MEDICINE; https://hdl.handle.net/10281/556892
DOI:	10.1056/NEJMoa1702752
Availability:	https://hdl.handle.net/10281/556892; https://doi.org/10.1056/NEJMoa1702752
Accession Number:	edsbas.C443583D
Database:	BASE