The case for eteplirsen: Paving the way for precision medicine

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Bibliographic Details
Published in:Molecular genetics and metabolism 2016-06, Vol.118 (2), p.70-71
Main Authors: Miceli, M. Carrie, Nelson, Stanley F.
Format: Article
Language:English
Subjects:
Administration, Intravenous
Child
Disease Progression
Dystrophin - genetics
Exons
Humans
Male
Morpholinos - genetics
Morpholinos - therapeutic use
Muscular Dystrophy, Duchenne - genetics
Muscular Dystrophy, Duchenne - therapy
Mutation
Open Reading Frames
Precision Medicine
Rare Diseases - genetics
Rare Diseases - therapy
RNA, Messenger - genetics
Treatment Outcome
United States
United States Food and Drug Administration
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2016.04.001