ArticleJAMA network open2020
Small Data Challenges of Studying Rare Diseases.
Article in JAMA network open, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 70 papers, 7 of them syntheses 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
70 citing papers in PubMed, 7 syntheses or guidelines pooled it.
- Prenatal opioid exposure, and health, social and educational outcomes for school-aged children: A systematic review.Addiction (Abingdon, England) · 2026Pooled it
- Exclusive human milk diet and mortality before discharge in premature and very low birthweight infants: a systematic review and meta-analysis.Frontiers in nutrition · 2026Pooled it
- Pooled it
- Capturing Real-World Rare Disease Patient Journeys: Are Current Methodologies Sufficient for Informed Healthcare Decisions?Journal of evaluation in clinical practice · 2025Pooled it
- Sexual health outcomes among adolescent and young adult cancer patients: a systematic review and meta-analysis.JNCI cancer spectrum · 2023Pooled it
- Barriers and facilitators to the implementation of guidelines in rare diseases: a systematic review.Orphanet journal of rare diseases · 2023Pooled it
- Necessity of Prophylactic Extrapleural Chest Tube During Primary Surgical Repair of Esophageal Atresia: A Systematic Review and Meta-Analysis.Frontiers in pediatrics · 2022Pooled it
- Feasibility and safety of sailing based rehabilitation for rare skeletal disorders using wearable sensors and patient reported outcomes.Scientific reports · 2025Trial
- A phase Ib/IIa clinical trial of dantrolene sodium in patients with Wolfram syndrome.JCI insight · 2021Trial
- Privacy-Preserving Framework for Multi-Institutional Medical Time-Series Analysis via Homomorphic Encryption: Design and Development Study.JMIR formative research · 2026Article
- Using Latent Representations to Link Disjoint Longitudinal Data for Mixed-Effects Regression.Statistics in medicine · 2026Article
- Identification of Nuclear Genetic Loci Linked to Clinical Features of the m.3243A>G Mitochondrial DNA Variant.Neurology. Genetics · 2026Article
- Vulnerability, protection and fairness: ethical and regulatory limits in small-N gene therapy trials.Trials · 2026Article
- RD-OMICS: An Integrative Multi-Omics Data Inventory in Rare Diseases.bioRxiv : the preprint server for biology · 2026Article
- Mandibular trabecular microarchitecture as a radiographic biomarker of bone health in children with 22q11.2 deletion syndrome.BMC pediatrics · 2026Article
- Psychometric properties of functional mobility outcome measures in children with arthrogryposis multiplex congenita.Developmental medicine and child neurology · 2026Article
- Unifying to Advance Understanding: Collaborative, Community-Driven and 'Open' Approaches for Better Science in Sport.Sports medicine (Auckland, N.Z.) · 2026Review
- A weakly supervised transformer for rare disease diagnosis and subphenotyping from EHRs with pulmonary case studies.NPJ digital medicine · 2026Article
- From potential to podium: what we still don't know about talent identification and development in Para athletics, a scoping review.Frontiers in sports and active living · 2026Review
- Navigating the landscape of personalized oncology: overcoming challenges and expanding horizons with computational modeling.Journal of the American Medical Informatics Association : JAMIA · 2026Article
10 more citing papers are in PubMed but not listed here.
Corrections and comments
- Comment on
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
PubMed holds no abstract for this paper.
Indexed as
Identifiers
32202640What 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.