Retrospective study of the natural history and genetic subtypes of ALS patients
Project Description: This project involved an analysis of the natural history and genetic subtypes of ALS patients using data extracted from Precision ALS partner sites
Project Objective: The common objective of the project was to research ALS natural history in patients, and their genetic subtypes using existing datasets held by the Precision ALS partner sites, combined into one harmonised dataset to enable new insights into ALS patient rate of functional decline, time to key clinical milestones, and survival and so provide valuable new information that will support new pharmaceutical developments for the clinical treatment of ALS patients. To examine this This requires collaborative inputs from the partners expertise in data science, clinical domain, and pharmaceutical outcomes to affect the full ambition of the project.
Project Status: Complete. This project has been successfully completed. The six questions which were set out at the outset of this study have been examined by the Precision ALS team through a set of coordinated analyses of the ‘Extant Dataset’ made up of ~21,000 individuals with ALS from the 9 participating Precision ALS sites. In addition to this a seventh article was prepared entitled ‘Challenges and solutions to complex data governance issues in cross-national, cross-sectoral, multidisciplinary real world health research’ to capture the experiences and expertise within the Precision ALS Consortium within this domain. All seven articles were published in a special supplement in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration in 2025. The details of these articles can be found on Our Publications page or by clicking on the image below.
