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• The proposed staging system is based on clinically meaningful end-points and has excellent construct validity • In this proposed staging system, individuals with CLN3 disease progress through each stage… Click to show full abstract
• The proposed staging system is based on clinically meaningful end-points and has excellent construct validity • In this proposed staging system, individuals with CLN3 disease progress through each stage of the disease as a function of age. • Elimination of the “Loss of Independence in ADLs stage from System 1 resulted in more discrete stages with little overlap. • A valid staging system has high potential for use in stratification for clinical trials and for development of stage-specific outcome measures. Stage 2 (First Seizure) and Stage 3 (Loss of Independence in ADLs) overlapped substantially. We eliminated Stage 3 because the First Seizure was a more discrete end-point.
Journal Title: Molecular Genetics and Metabolism
Year Published: 2018
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