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

BACKGROUND AND OBJECTIVES: The Pompe Disease Symptom Scale (PDSS) and Impact Scale (PDIS) were created to measure the severity of symptoms and functional limitations experienced by patients with late-onset Pompe disease (LOPD). The objectives of this analysis were to establish a scoring algorithm an...

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Detalles Bibliográficos
Autores principales: Dimachkie, Mazen M., Kishnani, Priya S., Ivanescu, Cristina, Flore, Giulio, Gwaltney, Chad, van der Beek, Nadine A.M.E., Hamed, Alaa, An Haack, Kristina, Pollissard, Laurence, Baranowski, Eileen, Sparks, Susan E., DasMahapatra, Pronabesh
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10409572/
https://www.ncbi.nlm.nih.gov/pubmed/37559825
http://dx.doi.org/10.1212/CPJ.0000000000200181

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