Gait, Balance, and Coordination Impairments in Niemann Pick Disease, Type C1

Title

Gait, Balance, and Coordination Impairments in Niemann Pick Disease, Type C1

Creator

Sansare A; Zampieri C; Alter K; Stanley C; Farhat N; Keener L A; Porter F

Publisher

Journal of Child Neurology

Date

2018

Subject

adolescent; retrospective study; priority journal; scoring system; school child; outcome assessment; preschool child; intervention study; human; article; child; female; male; adult; clinical article; young adult; comparative study; body equilibrium; stabilography; performance; neurologic examination; balance impairment; coordination disorder; gait disorder; Niemann Pick disease; 2 hydroxypropyl beta cyclodextrin; 94035-02-6 (2 hydroxypropyl beta cyclodextrin); case control study; clinical evaluation; disease severity assessment; human development; molecular stability; motion analysis system; NIH NPC Neurologic Severity Scale; research; spatiotemporal analysis; upper limb; vts 270; tone and motor problems; NPC; trajectory; characteristics

Description

This is the first study to objectively measure gait, balance, and upper limb coordination in a group of patients with NPC1 and compare the results to age and gender matched controls. This is also the first study to report effect sizes in these measures. Spatiotemporal gait analysis, static and dynamic posturography, and upper limb reaching motion analysis were performed. The findings showed that the NPC1 subjects had statistically significant deficits on 12 out of the 16 parameters investigated compared to controls, and large effect sizes for all but 1 parameter. When ranking the variables in terms of the effect sizes, the top 5 included at least 1 parameter from each of the 3 motor domains investigated. These results can provide insight to clinical researchers on the selection of outcome measures for longitudinal and interventional studies.

Rights

Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).

Citation

Sansare A; Zampieri C; Alter K; Stanley C; Farhat N; Keener L A; Porter F, “Gait, Balance, and Coordination Impairments in Niemann Pick Disease, Type C1,” Pediatric Palliative Care Library, accessed October 20, 2021, https://pedpalascnetlibrary.omeka.net/items/show/16799.

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