Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study.

Mercuri, Eugenio

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study. [electronic resource] - Journal of comparative effectiveness research 04 2020 - 341-360 p. digital

Publication Type: Journal Article; Multicenter Study; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.

2042-6313

10.2217/cer-2019-0171 doi


Codon, Nonsense--genetics
Dystrophin--genetics
Humans
Muscular Dystrophy, Duchenne--drug therapy
Oxadiazoles--adverse effects
Registries
Treatment Outcome