Clinical rating scale for pantothenate kinase‐associated neurodegeneration: A pilot study
Sign Up to like & getrecommendations! Published in 2017 at "Movement Disorders"
DOI: 10.1002/mds.27129
Abstract: Pantothenate kinase‐associated neurodegeneration is a progressive neurological disorder occurring in both childhood and adulthood. The objective of this study was to design and pilot‐test a disease‐specific clinical rating scale for the assessment of patients with… read more here.
Keywords: pantothenate kinase; clinical rating; kinase associated; associated neurodegeneration ... See more keywords
Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data
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DOI: 10.1371/journal.pone.0302382
Abstract: Background The CLN2 Clinical Rating Scale evaluates disease progression in CLN2 disease, an ultra-rare, neurodegenerative disorder with late infantile onset. To validate the Clinical Rating Scale, a comparison with the Pediatric Quality of Life Inventory… read more here.
Keywords: disease; clinical rating; pedsql; scale ... See more keywords