Patient-reported outcomes and quality of life in PMM2-CDG
Anna N. Ligezka, Anab Mohamed, Carlota Pascoal, Vanessa dos Reis Ferreira, Suzanne Boyer, Christina Lam, Andrew C. Edmondson, Wirginia Krzyściak, Raphael Golebiowski, Judit Perez Ortiz, Éva Morava
Topics & Concepts
MedicineQuality of life (healthcare)CohortAnxietyPhysical therapyPopulationDiseaseNatural history studyPatient-Reported Outcomes Measurement Information SystemRating scaleInternal medicinePsychometricsClinical psychologyPsychiatryPsychologyComputerized adaptive testingNursingEnvironmental healthDevelopmental psychologyLysosomal Storage Disorders ResearchGenomics and Rare DiseasesGenomic variations and chromosomal abnormalities