The case for eteplirsen: Paving the way for precision medicine

MeSH terms

• 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*
• RNA, Messenger / genetics
• Rare Diseases / genetics
• Rare Diseases / therapy*
• Treatment Outcome
• United States
• United States Food and Drug Administration