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Muscle function and patient-reported outcomes
Muscle function testing and patient-reported outcomes were assessed. (A) 6MWT, (B) FVC, (C) FVC in the supine position, (D) Fatigue Severity Scale, (E) PROMIS Fatigue Scale, and (F) Rasch-Built Pompe Specific Activity Scale. Statistical significance analyzed by paired t test, ns = not significant. # indicates that ERT was discontinued at week 26.
