No abstract available
MeSH terms
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Administration, Intravenous
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Child
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Disease Progression
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Dystrophin / genetics
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Exons
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Humans
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Male
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Morpholinos / genetics
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Morpholinos / therapeutic use*
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Muscular Dystrophy, Duchenne / genetics
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Muscular Dystrophy, Duchenne / therapy*
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Mutation
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Open Reading Frames
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Precision Medicine*
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RNA, Messenger / genetics
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Rare Diseases / genetics
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Rare Diseases / therapy*
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Treatment Outcome
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United States
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United States Food and Drug Administration
Substances
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Dystrophin
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Morpholinos
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RNA, Messenger
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eteplirsen