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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

par Finkel, Richard S;Mercuri, Eugenio;Darras, Basil T;Connolly, Anne M;Kuntz, Nancy L;Kirschner, Janbernd;Chiriboga, Claudia A;Saito, Kayoko;Deconinck, Nicolas ;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.;ENDEAR Study Group,
Référence The New England journal of medicine, 377, 18, page (1723-1732)
Publication Publié, 2017

Article révisé par les pairs

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Titre:	Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.
Auteur:	Finkel, Richard S; Mercuri, Eugenio; Darras, Basil T; Connolly, Anne M; Kuntz, Nancy L; Kirschner, Janbernd; Chiriboga, Claudia A; Saito, Kayoko; Deconinck, Nicolas; 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.; ENDEAR Study Group,
Informations sur la publication:	The New England journal of medicine, 377, 18, page (1723-1732)
Statut de publication:	Publié, 2017
Sujet CREF:	Sciences bio-médicales et agricoles
MeSH keywords:	Age of Onset
	Disease-Free Survival
	Double-Blind Method
	Female
	Humans
	Infant
	Injections, Spinal
	Male
	Motor Skills
	Oligonucleotides -- adverse effects -- therapeutic use
	Oligonucleotides, Antisense -- adverse effects -- therapeutic use
	RNA, Messenger -- drug effects -- metabolism
	Respiration, Artificial
	Spinal Muscular Atrophies of Childhood -- drug therapy -- genetics -- mortality -- physiopathology
	Survival Analysis
	Survival of Motor Neuron 2 Protein -- genetics -- metabolism
Langue:	Anglais
Identificateurs:	urn:issn:0028-4793
	info:doi/10.1056/NEJMoa1702752
	info:pmid/29091570