Natural history and biomarker study of multisystem proteinopathy (MSP), most often caused by pathogenic variants in the VCP gene.
This study aims to develop clinical outcome measures and identify biomarkers for use in clinical trials.
Historical note: Our observation that ALS is one of the phenotypic manifestations of a disorder previously called “IBMPFD”, with TDP-43 being the underlying pathology, led us to propose renaming this disorder as “MSP” in 2013.
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