Establishing how much improvement in lung function and distance walked is clinically important for adult patients with Pompe disease.

Aglina LikaEleni-Rosalina AndrinopoulouNadine A M E van der BeekDimitris RizopoulosAns T van der Ploeg Michelle E Kruijshaar

Published in: European journal of neurology (2024)

and 6MWT derived in this study can be used to interpret differences between and within groups of patients with Pompe disease in clinical trials and cohort studies.

Keyphrases

lung function
late onset
clinical trial
replacement therapy
cystic fibrosis
chronic obstructive pulmonary disease
air pollution
early onset
open label
smoking cessation