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

The Lancet (British edition), 2016-12, Vol.388 (10063), p.3017-3026 [Peer Reviewed Journal]

Elsevier Ltd ;2016 Elsevier Ltd ;Copyright © 2016 Elsevier Ltd. All rights reserved. ;Copyright Elsevier Limited Dec 17, 2016 ;ISSN: 0140-6736 ;EISSN: 1474-547X ;DOI: 10.1016/S0140-6736(16)31408-8 ;PMID: 27939059 ;CODEN: LANCAO

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