ArticleNeurology2011
Quantitative telemedicine ratings in Batten disease: implications for rare disease research.
Article in Neurology, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled 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.
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.
Who cites it
18 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Recommendations for the diagnosis and management of cln3 disease (batten disease) using the Delphi consensus methodology.Orphanet journal of rare diseases · 2026Guideline
- Trial
- Speech, Language and Non-verbal Communication in CLN2 and CLN3 Batten Disease.Journal of inherited metabolic disease · 2025Article
- What role can decentralized trial designs play to improve rare disease studies?Orphanet journal of rare diseases · 2022Article
- The Impact of the COVID-19 Pandemic on the Perception of Health and Treatment-Related Issues among Patients with Phenylketonuria in Poland-The Results of a National Online Survey.International journal of environmental research and public health · 2021Article
- Practicing in a Pandemic: A Clinician's Guide to Remote Neurologic Care.Neurology. Clinical practice · 2021Article
- Rare disease care pathways in the EU: from odysseys and labyrinths towards highways.Journal of community genetics · 2021Article
- Video research visits for atypical parkinsonian syndromes among Fox Trial Finder participants.Neurology. Clinical practice · 2020Article
- Therapeutic landscape for Batten disease: current treatments and future prospects.Nature reviews. Neurology · 2019Review
- The Promise of Telemedicine for Movement Disorders: an Interdisciplinary Approach.Current neurology and neuroscience reports · 2018Review
- Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions.Molecular genetics and metabolism · 2016Article
- Remote Assessment of Cognitive Function in Juvenile Neuronal Ceroid Lipofuscinosis (Batten disease): A Pilot Study of Feasibility and Reliability.Journal of child neurology · 2016Article
- Novel methods and technologies for 21st-century clinical trials: a review.JAMA neurology · 2015Review
- Clinical trials in rare disease: challenges and opportunities.Journal of child neurology · 2013Article
- Neurobehavioral features and natural history of juvenile neuronal ceroid lipofuscinosis (Batten disease).Journal of child neurology · 2013Review
- Methodology of clinical research in rare diseases: development of a research program in juvenile neuronal ceroid lipofuscinosis (JNCL) via creation of a patient registry and collaboration with patient advocates.Contemporary clinical trials · 2013Article
- Reliability and validity of the Wolfram Unified Rating Scale (WURS).Orphanet journal of rare diseases · 2012Article
- Understanding dentatorubral-pallidoluysian atrophy (DRPLA) symptoms and impacts on daily life: a qualitative interview study with patients and caregivers.Therapeutic advances in rare diseaseArticle
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Authors and funding
6 authors.
Funding
Abstract
objectiveTo determine if remote administration of the Unified Batten Disease Rating Scale (UBDRS) Physical Impairment subscale by telemedicine is reliable and feasible across a broad range of disease severity.
methodsFor the majority (n = 10) of subjects, the examination was performed by a nonphysician who had been trained to perform the examination but not to score the subjects. A trained rater scored the subjects via live video; a second trained rater performed a separate examination in person and scored that examination. For 3 telemedicine evaluations, examinations were performed and scored by a trained rater while a second trained rater simultaneously scored the subjects via live video. Reliability was determined by intraclass correlation coefficient (ICC).
resultsSubjects (n = 13) represented a wide range of disease severity. Remote administration of the UBDRS Physical Impairment subscale had high interrater reliability across all subjects (ICC = 0.94). When only the subjects (n = 10) who had been examined by the nonphysician and scored remotely were included in the analysis, the reliability was unchanged (ICC = 0.95).
conclusionsThe UBDRS Physical Impairment subscale is reliable and feasible for remote administration. Telemedicine has the potential to be a useful tool in rare neurologic disease research and clinical assessment.
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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.