Abstract
No funding contributed to the writing of this commentary. Brandsema reports consulting for Alexion, Audentes, AveXis, Biogen, Cytokinetics, PTC Therapeutics, Sarepta, and WaVe and has received research funding as a site investigator from Alexion, AveXis, Biogen, CSL Behring, Cytokinetics, Fibrogen, Pfizer, PTC Therapeutics, Sarepta, Summit, and WaVe.
MeSH terms
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Cost-Benefit Analysis
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Humans
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Insurance Coverage / economics*
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Insurance, Pharmaceutical Services / economics*
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Insurance, Pharmaceutical Services / legislation & jurisprudence
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Morpholinos / economics
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Morpholinos / therapeutic use*
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Muscular Dystrophy, Duchenne / drug therapy*
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Muscular Dystrophy, Duchenne / economics
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Oligonucleotides / economics
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Oligonucleotides / therapeutic use*
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Pregnenediones / economics
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Pregnenediones / therapeutic use*
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United States
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United States Food and Drug Administration / legislation & jurisprudence
Substances
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Morpholinos
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Oligonucleotides
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Pregnenediones
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golodirsen
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eteplirsen
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deflazacort