Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
University College London · Newcastle University · +6 more institutions
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Abstract
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Citation impact
859
total citations
- FWCI
- 40.70
- Percentile
- 100%
- References
- 41
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Authors
20Topics & keywords
Topics
Keywords
- Duchenne muscular dystrophy
- Dystrophin
- Morpholino
- Medicine
- Tolerability
- Exon skipping
- Adverse effect
- mdx mouse
UN Sustainable Development Goals
- Good health and well-being
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