Study on the quality of life of women
affected by X-linked adrenoleukodystrophy [ALD]
A new study is available on Leuconnect!
In partnership with ELA International and under the responsibility of Professor Wolfgang Koehler in Germany, the team from the University of Leipzig is opening a study on the quality of life of women affected by the mutation of the ABCD1 gene, responsible for X-linked adrenoleukodystrophy.
This study is aimed at women affected by X-linked adrenoleukodystrophy.It is now known that women with this mutation can develop late symptoms that progress with age, but these are often underestimated due to the more moderate and delayed progression of the disease.
This study should make it possible to identify the influence of various neurological symptoms on the quality of life of these patients and to highlight the resulting difficulties. It could speed up the diagnosis of the disease and lead to new preventive treatments.
This study is being conducted on the Leuconnect platform
The study will be conducted using online questionnaires on Leuconnect. They will cover a variety of areas, including activities of daily living.
This study will be conducted in 3 languages, and is aimed at English, French and German speakers.
If you have not already done so, come and discover the platform and join the cohort now by registering on Leuconnect at the following link: https://www.leuconnect.com
The first study on the impact of metachromatic leukodystrophy (MLD) on the lives of families is open on Leuconnect! Completed
In partnership with ELA International, and joined by ARGO Santé, a health research and consulting firm, ORCHARD Therapeutics is launching a study on the experiences of parents of children with MLD who have not been treated with gene therapy. The objective of this study is to better understand the evolution of the disease and to measure the impact of the disease on the quality of life and its impact on the daily lives of families.
This study is aimed at families affected by metachromatic leukodystrophy residing in France. It has already been carried out in other countries and is part of a broader approach.
It will take place in two stages, the first stage being a questionnaire addressed to the parents who will be included in this study, and then an interview that will allow the anonymous collection of family testimonies.
This study is being conducted on the Leuconnect platform.
If you haven’t already done so, come and discover the platform and join the cohort now by registering on Leuconnect at the following link:
Educational videos and “practical” articles are available on the platform to support you in your approach.
General information on the study is available to all by visiting the Leuconnect news page or by clicking directly on this link: MLD Study
Because the expert assessment of families impacted by leukodystrophies is essential for research, it is important to be part of the Leuconnect cohort, to participate in online studies and to keep in touch with the community.
This first online study paves the way for other studies already in preparation and soon to be made available.