Evidence map›Paper›PMID 29246094›Full record

Trial reportJournal of child neurology2018

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

Ashwini Sansare, Cris Zampieri, Katharine Alter, Christopher Stanley, Nicole Farhat, Lee Ann Keener, Forbes Porter

Abstract readClinical Trial, Phase IClinical Trial, Phase II
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of child neurology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
0.3field-weighted citation impact, top 37% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed, 3 citations in OpenAlex.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 2 institutions in 1 country.

Ashwini Sansare1 Functional and Applied Biomechanics Section, Rehabilitation Medicine Department, National Institutes of Health Clinical Center, Bethesda, MD, USA.ORCID http://orcid.org/0000-0002-3218-1649
Cris Zampieri1 Functional and Applied Biomechanics Section, Rehabilitation Medicine Department, National Institutes of Health Clinical Center, Bethesda, MD, USA.
Katharine Alter1 Functional and Applied Biomechanics Section, Rehabilitation Medicine Department, National Institutes of Health Clinical Center, Bethesda, MD, USA.
Christopher Stanley1 Functional and Applied Biomechanics Section, Rehabilitation Medicine Department, National Institutes of Health Clinical Center, Bethesda, MD, USA.
Nicole Farhat2 Section on Molecular Dysmorphology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
Lee Ann Keener3 Nursing Department, National Institutes of Health Clinical Center, Bethesda, MD, USA.
Forbes Porter2 Section on Molecular Dysmorphology, Eunice Kennedy Shriver National Institute of Child Health and Human Development, Bethesda, MD, USA.
National Institutes of Health Clinical Center · USEunice Kennedy Shriver National Institute of Child Health and Human Development · US

Funding

Clinical Investigations of Niemann-Pick Disease, type CZIAHD008824 · NICHD · EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH & HUMAN DEVELOPMENT · PI PORTER, FORBES · 2009 to 2019
$5.2M
Intramural NIH HHS ZIA HD008824
6 · The paper itself

Abstract

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.

Indexed as

GaitMotor SkillsPostural BalanceAdolescentBiomechanical PhenomenaCase-Control StudiesChildChild, PreschoolFemaleHumansMaleNiemann-Pick Disease, Type CSeverity of Illness IndexUpper ExtremityYoung Adultataxiachildrendevelopmental disabilitylysosomal diseaserehabilitation

Identifiers

PMID29246094
PMCPMC6534353
OpenAlexW2773368442

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.