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Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Journal article   Open access  Peer reviewed

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Richard S Finkel, Eugenio Mercuri, Basil T Darras, Anne M Connolly, Nancy L Kuntz, Janbernd Kirschner, Claudia A Chiriboga, Kayoko Saito, Laurent Servais, Eduardo Tizzano, …
The New England journal of medicine, Vol.377(18), pp.1723-1732
2017-11-02
PMID: 29091570

Abstract

Injections, Spinal Spinal Muscular Atrophies of Childhood - genetics Double-Blind Method Oligonucleotides - therapeutic use Humans Spinal Muscular Atrophies of Childhood - drug therapy Infant Male Oligonucleotides - adverse effects Oligonucleotides, Antisense - adverse effects RNA, Messenger - metabolism Survival of Motor Neuron 2 Protein - metabolism Respiration, Artificial Disease-Free Survival Oligonucleotides, Antisense - therapeutic use Survival of Motor Neuron 2 Protein - genetics Age of Onset Survival Analysis Female Spinal Muscular Atrophies of Childhood - mortality Spinal Muscular Atrophies of Childhood - physiopathology Motor Skills RNA, Messenger - drug effects
url
https://doi.org/10.1056/NEJMoa1702752View
Published (Version of record) Open

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Citation topics
1 Clinical & Life Sciences
1.255 Musculoskeletal Disorders
1.255.2204 Spinal Muscular Atrophy
Web Of Science research areas
Clinical Neurology
ESI research areas
Clinical Medicine

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