关键词: STRIDE Registry ataluren dystrophin effectiveness nonsense mutation Duchenne muscular dystrophy safety
Mesh : Codon, Nonsense / genetics Dystrophin / genetics Humans Muscular Dystrophy, Duchenne / drug therapy genetics Oxadiazoles / adverse effects therapeutic use Registries Treatment Outcome
来 源:
DOI:10.2217/cer-2019-0171
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