ArticleOrphanet journal of rare diseases2012
Reliability and validity of the Wolfram Unified Rating Scale (WURS).
Article in Orphanet journal of rare diseases, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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Who cites it
21 citing papers in PubMed, 24 citations in OpenAlex.
- A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome.JCI insight · 2021Trial
- Neuroimaging evidence of deficient axon myelination in Wolfram syndrome.Scientific reports · 2016Trial
- Neurofilament light chain but not glial fibrillary acidic protein serum levels are elevated in Wolfram syndrome.Frontiers in neuroscience · 2026Review
- Article
- Clinical trials for Wolfram syndrome neurodegeneration: Novel design, endpoints, and analysis models.PloS one · 2025Article
- Genotype and Clinical Characteristics of Patients with Wolfram Syndrome and WFS1-related Disorders.medRxiv : the preprint server for health sciences · 2023Article
- Genotype and clinical characteristics of patients with Wolfram syndrome and WFS1-related disorders.Frontiers in genetics · 2023Article
- Longitudinal progression of diabetes mellitus in Wolfram syndrome: The Washington University Wolfram Research Clinic experience.Pediatric diabetes · 2022Observational
- Plasma Neurofilament Light Chain Levels Are Elevated in Children and Young Adults With Wolfram Syndrome.Frontiers in neuroscience · 2022Article
- Urinary Tract Involvement in Wolfram Syndrome: A Narrative Review.International journal of environmental research and public health · 2021Review
- Content Validation of Clinician-Reported Items for a Severity Measure for CDKL5 Deficiency Disorder.Journal of child neurology · 2021Article
- Sleep disturbances in Wolfram syndrome.Orphanet journal of rare diseases · 2019Article
- Evidence for altered neurodevelopment and neurodegeneration in Wolfram syndrome using longitudinal morphometry.Scientific reports · 2019Article
- Pancreatic stone protein/regenerating protein is a potential biomarker for endoplasmic reticulum stress in beta cells.Scientific reports · 2019Article
- Understanding activity participation among individuals with Wolfram Syndrome.The British journal of occupational therapy · 2018Article
- Visual pathway function and structure in Wolfram syndrome: patient age, variation and progression.BMJ open ophthalmology · 2018Article
- The effects of disease-related symptoms on daily function in Wolfram Syndrome.Translational science of rare diseases · 2017Article
- Selective cognitive and psychiatric manifestations in Wolfram Syndrome.Orphanet journal of rare diseases · 2015Article
- Ophthalmologic correlates of disease severity in children and adolescents with Wolfram syndrome.Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus · 2014Article
- Phenotypic characteristics of early Wolfram syndrome.Orphanet journal of rare diseases · 2013Article
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 1 country.
Funding
Abstract
backgroundWolfram syndrome (WFS) is a rare, neurodegenerative disease that typically presents with childhood onset insulin dependent diabetes mellitus, followed by optic atrophy, diabetes insipidus, deafness, and neurological and psychiatric dysfunction. There is no cure for the disease, but recent advances in research have improved understanding of the disease course. Measuring disease severity and progression with reliable and validated tools is a prerequisite for clinical trials of any new intervention for neurodegenerative conditions. To this end, we developed the Wolfram Unified Rating Scale (WURS) to measure the severity and individual variability of WFS symptoms. The aim of this study is to develop and test the reliability and validity of the Wolfram Unified Rating Scale (WURS).
methodsA rating scale of disease severity in WFS was developed by modifying a standardized assessment for another neurodegenerative condition (Batten disease). WFS experts scored the representativeness of WURS items for the disease. The WURS was administered to 13 individuals with WFS (6-25 years of age). Motor, balance, mood and quality of life were also evaluated with standard instruments. Inter-rater reliability, internal consistency reliability, concurrent, predictive and content validity of the WURS were calculated.
resultsThe WURS had high inter-rater reliability (ICCs>.93), moderate to high internal consistency reliability (Cronbach's α = 0.78-0.91) and demonstrated good concurrent and predictive validity. There were significant correlations between the WURS Physical Assessment and motor and balance tests (rs>.67, p<.03), between the WURS Behavioral Scale and reports of mood and behavior (rs>.76, p<.04) and between WURS Total scores and quality of life (rs=-.86, p=.001). The WURS demonstrated acceptable content validity (Scale-Content Validity Index=0.83).
conclusionsThese preliminary findings demonstrate that the WURS has acceptable reliability and validity and captures individual differences in disease severity in children and young adults with WFS.
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Registered trials
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.