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

Document Type

Article

Author

Finkel, Richard S; Chiriboga, Claudia A; Vajsar, Jiri; Day, John W; Montes, Jacqueline; De Vivo, Darryl C; Yamashita, Mason; Rigo, Frank; Hung, Gene; Schneider, Eugene; Norris, Daniel A; Xia, Shuting; Bennett, C Frank; Bishop, Kathie M

Source

In The Lancet 17 December 2016-6 January 2017 388(10063):3017-3026

Subject

Primary Research
Articles

Language

ISSN

0140-6736

Online Access

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