Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients - McDonald, Craig M; Muntoni, Francesco; Penematsa, Vinay; Jiang, Joel; Kristensen, Allan; Bibbiani, Francesco; Goodwin, Elizabeth; Gordish-Dressman, Heather; Morgenroth, Lauren; Werner, Christian; Li, James; Able, Richard; et al. (Journal of comparative effectiveness research, 2022)
Keywords: ataluren; benefits; Clinical Medicine; Codon, Nonsense; diagnosis; dmd; Duchenne muscular dystrophy; dystrophin; efficacy; glucocorticoids; Health Care Sciences & Services; Humans; Klinisk medicin; life; loss of ambulation; management; Muscular Dystrophy, Duchenne - genetics; natural-history; nonsense mutation; nonsense mutation Duchenne muscular dystrophy; Oxadiazoles - therapeutic use; progression; respiratory function; Study 019; Walking; nonsense mutation Duchenne muscular dystrophy; ataluren; loss of ambulation; respiratory function; dystrophin; efficacy; Study 019