Mole, SE;
Gardner, E;
Mason, HL;
(2025)
How can real-world data, registries, and databases address the challenges of rare diseases?
Medical Writing
, 34
(1)
pp. 46-50.
10.56012/kwle5627.
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Abstract
A goal for all diseases is a treatment that works to prevent, halt, or reverse their effects – essentially, a cure. Achieving this requires early diagnosis, knowledge of disease mechanisms, and effective treatment. For rare diseases, each of these elements is a huge challenge. This perspective explores how real-world and registry data can address these obstacles and considers future developments with the potential for the most significant impact.
| Type: | Article |
|---|---|
| Title: | How can real-world data, registries, and databases address the challenges of rare diseases? |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.56012/kwle5627 |
| Publisher version: | https://doi.org/10.56012/kwle5627 |
| Language: | English |
| Additional information: | This version is the version of record. For information on re-use, please refer to the publisher’s terms and conditions. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Genetics and Genomic Medicine Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10208449 |
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