Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data

• Main Authors: Priya S. Kishnani (Author), Simon Shohet (Author), Syed Raza (Author), Noemi Hummel (Author), Jeffrey P. Castelli (Author), Sheela Sitaraman Das (Author), Heng Jiang (Author), Agnieszka Kopiec (Author), Ian Keyzor (Author), Andreas Hahn (Author)
• Format: Book
• Published: SpringerOpen, 2024-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background The construct validity and interpretation of the Patient-Reported Outcome Measurement Information System (PROMIS®) Physical Function short form 20a (PF20a) questionnaire were evaluated for patients with late-onset Pompe disease (LOPD), a rare, autosomal recessive, progressive neu...