Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS ® ) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data.
Priya S KishnaniSimon ShohetSyed RazaNoemi HummelJeffrey P CastelliSheela Sitaraman DasHeng JiangAgnieszka KopiecIan KeyzorAndreas HahnPublished in: Journal of patient-reported outcomes (2024)
gov/search?term=NCT03729362 .