With the mobilization of the families, health professionals and ELA, and under the lead of the firm ARGO Santé, a study on the impact of cerebral adrenoleukodystrophy on the quality of life of patients and their families was conducted on Leuconnect. The platform allowed the expected recruitment and a rapid implementation of the study.
This survey of families explores the path of care and the impact of illness on their daily lives through quality of life questionaries and qualitative interviews.
The work of the teams of ELA, Caroline Sevin and Elise Yazbeck, neuropediatricians from Bicêtre hospital, Marieke Podevin and Gaëlle Thomas from ARGO Santé, and Fabrizia Bignani and Aurore Clément of Bluebird Bio France, was published in the peer reviewed scientific journal « Rare Disease and Orphan Drugs Journal » (RDODJ), demonstrating the quality of the work and the robustness of the results: Scientific Article “RDODJ”.
These results are now accessible to the international scientific community and will be a reference on the subject. They provide a better understanding of the impact of the disease on the quality of life of patients and their families, leading to symptomatic treatments and better management of the disease.
The first results of the study are available on Leuconnect:
Results of the study “impact of cALD on the daily lives of families”
For more than 10 years, ELA has invested massively in ALD research, and supports all initiatives that can change the lives of patients.
ELA warmly thanks the families who participated in this study for their valuable involvement in understanding the disease and its management.
