ReviewCold Spring Harbor perspectives in medicine2023
The Importance of Natural History Studies in Inherited Retinal Diseases.
Review in Cold Spring Harbor perspectives in medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
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
9 citing papers in PubMed.
- Natural History of Macular Sensitivity in Best Vitelliform Macular Dystrophy: Microperimetry-Derived Outcome Measures in Preparation For Clinical Trials.Investigative ophthalmology & visual science · 2026Article
- The Science of Rod-Mediated Dark Adaptation, an Outcome Measure for Age-Related Macular Degeneration: The 2025 Proctor Medal Lecture.Investigative ophthalmology & visual science · 2026Article
- Exploration and Application of Multiluminance Mobility Test in Bietti Crystalline Dystrophy.Translational vision science & technology · 2026Article
- Novel Genotype-Phenotype Correlations inOphthalmology science · 2026Article
- Natural History of Visual Function Changes Over Three Years in Normal Aging and in Early and Intermediate Age-Related Macular Degeneration: ALSTAR2.Translational vision science & technology · 2026Article
- Gene augmentation therapy restores vision and preserves photoreceptors in a mouse model of CNGA1-retinitis pigmentosa.Communications medicine · 2025Article
- Assessing Contrast Sensitivity Function in CRB1-Retinopathies: Exploring Child-Friendly Measures of Visual Function.Translational vision science & technology · 2024Article
- Key challenges in developing a gene therapy for Usher syndrome: machine-assisted scoping review.Journal of community genetics · 2024Article
- Endpoints and Design for Clinical Trials in USH2A-Related Retinal Degeneration: Results and Recommendations From the RUSH2A Natural History Study.Translational vision science & technology · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Natural history studies of inherited retinal diseases (IRDs) play a critical role in the design and implementation of treatment trials. Study objectives ideally encompass (1) understanding the time course and pattern of disease progression, (2) within genotypic and phenotypic subtypes of patient populations, and (3) characterizing a range of measures of vision function, retinal structure, and functional vision that may serve as endpoints. In rare disease, data quality standards are paramount to optimizing smaller sample sizes, including a prospective, standardized, and longitudinal approach to data collection. Multicenter studies additionally facilitate strength in numbers and generalizability, and multidisciplinary collaboration ensures a holistic approach to study design and knowledge-building. Dissemination of natural history study results, data sets, and lessons learned will stimulate further innovation and progress in IRD therapeutic research, including setting up future trial designs for their best chance of success.
Indexed as
Identifiers
What OpenQuestion holds
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.