Measurement Properties of 2 Novel PROs, the Pompe Disease Symptom Scale and Pompe Disease Impact Scale, in the COMET Study.

Mazen M DimachkiePriya S KishnaniCristina IvanescuGiulio FloreChad GwaltneyNadine A M E van der BeekAlaa HamedKristina An HaackLaurence PollissardEileen BaranowskiSusan E SparksPronabesh DasMahapatranull null

Published in: Neurology. Clinical practice (2023)

ClinicalTrials.gov identifier: NCT02782741.

Keyphrases

late onset
replacement therapy
early onset
patient reported