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Comment in: FDA Approval of Eteplirsen for Muscular Dystrophy-Reply. [JAMA. 2017] Comment on: Approving a Problematic Muscular Dystrophy Drug: Implications for FDA Policy. [JAMA. 2016]Article
Kaifer KA, Author ; Villalon E ; Osman EY ; Glascock JJ ; Arnold LL ; Cornelison DD ; Lorson CL | 2017Article
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Nakamura A, Author ; Shiba N ; Miyazaki D ; Nishizawa H ; Inaba Y ; Fueki N ; Maruyama R ; Echigoya Y ; Yokota T | 2017Article
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Echigoya Y, Author ; Lim KRQ ; Trieu N ; Bao B ; Miskew Nichols B ; Vila MC ; Novak JS ; Hara Y ; Lee J ; Touznik A ; Mamchaoui K ; Aoki Y ; Takeda S ; Nagaraju K ; Mouly V ; Maruyama R ; Duddy W ; Yokota T | 2017Article
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Novak JS, Author ; Hogarth MW ; Boehler JF ; Nearing M ; Vila MC ; Heredia R ; Fiorillo AA ; Zhang A ; Hathout Y ; Hoffman EP ; Jaiswal JK ; Nagaraju K ; Cirak S ; Partridge TA | 2017Article
Tasfaout H, Author ; Buono S ; Guo S ; Kretz C ; Messaddeq N ; Booten S ; Greenlee S ; Monia BP ; Cowling BS ; Laporte J | 2017Article
Adams L, Author | 2017Comment on: Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. [Lancet. 2016]Article
Goemans N, Author ; Tulinius M ; Kroksmark AK ; Wilson R ; van den Hauwe M ; Campion G | 2017