ArticleNature biotechnology2014
Expanding rare disease drug trials based on shared molecular etiology.
Article in Nature biotechnology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 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
11 citing papers in PubMed.
- The Placebo Effect in Rare Disease Clinical Trials: Measurement, Impact, and Statistical Approaches for Patient-as-Own-Control Designs.Clinical and translational science · 2026Review
- Targeting shared molecular etiologies to accelerate drug development for rare diseases.EMBO molecular medicine · 2023Review
- New Developments and Possibilities in Reanalysis and Reinterpretation of Whole Exome Sequencing Datasets for Unsolved Rare Diseases Using Machine Learning Approaches.International journal of molecular sciences · 2022Review
- Article
- Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination.Frontiers in pharmacology · 2022Article
- Opportunities and challenges in translational science.Clinical and translational science · 2021Review
- Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.Nature reviews. Drug discovery · 2020Review
- Computational derivation of a molecular framework for hair follicle biology from disease genes.Scientific reports · 2017Article
- The context for the thematic grouping of rare diseases to facilitate the establishment of European Reference Networks.Orphanet journal of rare diseases · 2016Article
- Gene Therapy: The View from NCATS.Human gene therapy · 2016Article
- Looking Forward to New Therapies: A Personal Perspective on the Translational Landscape for Muscular Dystrophies.Journal of neuromuscular diseases · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
Abstract
PubMed holds no abstract for this paper.
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.