ReviewGene therapy2017
Building the patient community.
Review in Gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 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
5 citing papers in PubMed.
- People with Early Onset Parkinson's Disease: Empowered to Improve Care.Journal of Parkinson's disease · 2023Article
- 'Advocacy groups are the connectors': Experiences and contributions of rare disease patient organization leaders in advanced neurotherapeutics.Health expectations : an international journal of public participation in health care and health policy · 2022Article
- Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey.BMJ open · 2021Article
- Multiple Sulfatase Deficiency: A Disease Comprising Mucopolysaccharidosis, Sphingolipidosis, and More Caused by a Defect in Posttranslational Modification.International journal of molecular sciences · 2020Review
- Meet the researchers: an alternative method of engaging patients with research in mesothelioma.Research involvement and engagement · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
There are many challenges in conducting rare disease research. The conditions are often poorly understood, small patient populations are dispersed around the world, and there are limited funding opportunities. Patient groups can serve as a key partner in overcoming these challenges, as they understand the impact of rare conditions on patients' lives. This gives patient groups valuable scientific insights into the disease. This can be used to create research strategies, address research bottlenecks and directly fund research that appropriately addresses patient needs. Patient groups can also play a critical role in recruiting and retaining patients for clinical trials, which reduces time and resource waste. By partnering with patient groups, research teams can improve efficiency of research and best meet the needs of patients. Researchers can also play an important role in building and supporting patient groups to unlock these benefits.
Indexed as
Identifiers
28467403What 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.