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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