Delays in pulmonary decline in eteplirsen-treated patients with Duchenne muscular dystrophy.

Joel IffCharles GerritsYi ZhongEdward TuttleErica BirkYeya ZhengXander PaulErik K HenricsonCraig M McDonaldnull null

Published in: Muscle & nerve (2022)

The attenuation of FVC%p decline suggests that eteplirsen-treated patients had statistically significant and clinically meaningful attenuations in pulmonary decline compared with SoC patients.

Keyphrases

duchenne muscular dystrophy
end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
prognostic factors
peritoneal dialysis
patient reported outcomes