Validity and Reliability of Clinical and Patient‐Reported Outcomes in Multisystem Proteinopathy 1

Validity and Reliability of Clinical and Patient‐Reported Outcomes in Multisystem Proteinopathy 1

ABSTRACT Objective Valosin‐containing protein (VCP)‐associated multisystem proteinopathy 1 (MSP1) is caused by variants in the VCP gene. MSP1 results in various phenotypes including progressive myopathy, Paget's disease of bone, frontotemporal dementia, amyotrophic lateral sclerosis, and parkin...

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Main Authors: Lindsay N. Alfano, Megan A. Iammarino, Natalie F. Reash, Linda P. Lowes, Lindsay Pietruszewski, Kathleen Adderley, Lauren Humphrey, Audrey B. Knight, Christopher L. Steiner, Melissa A. Smith, Zarife Sahenk, Anne M. Connolly, Momen Almomen, Eleonora S. D'Ambrosio, Nathan Peck, Allison Peck
Format: Article
Language:English
Published: Wiley 2025-07-01
Series:Annals of Clinical and Translational Neurology
Subjects:
function
natural history
VCP‐associated multisystem proteinopathy
Online Access:https://doi.org/10.1002/acn3.70064
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