Onasemnogene Abeparvovec Treatment after Nusinersen in an Infant with Spinal Muscular Atrophy Type 1
Author:
Affiliation:
1. Department of Pediatrics and Child Health, Kurume University School of Medicine
2. Department of Pediatrics, Oita University Faculty of Medicine
Publisher
Kurume Medical Journal
Link
https://www.jstage.jst.go.jp/article/kurumemedj/69/3.4/69_MS6934008/_pdf
Reference13 articles.
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2. 2. Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology 2014; 83:810-817.
3. 3. Rigo F, Hua Y, Krainer AR, and Bennett CF. Antisense-based therapy for the treatment of spinal muscular atrophy. J Cell Biol 2012; 199:21-25.
4. 4. Lowes LP, Alfano LN, Arnold WD, Shell R, Prior TW et al. Impact of age and motor function in a phase 1/2A study of infants with SMA type 1 receiving single-dose gene replacement therapy. Pediatr Neurol 2019; 98:39-45.
5. 5. Sivaramakrishnan M, McCarthy KD, Campagne S, Huber S, Meier S et al. Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers. Nat Commun 2017; 8:1476.
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