Improvements in health status and utility associated with ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy.

Landfeldt, Erik

Improvements in health status and utility associated with ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy. [electronic resource] - Muscle & nerve 03 2020 - 363-368 p. digital

Publication Type: Journal Article

1097-4598

10.1002/mus.26787 doi


Codon, Nonsense
Delphi Technique
Health Status
Humans
Muscular Dystrophy, Duchenne--drug therapy
Oxadiazoles--therapeutic use
Treatment Outcome