Stay up to date
16th January 2025
Edge Hill University is working with NHS clinicians to develop national education standards and resources for sick day/emergency management of adrenal insufficiency in children and young people. They are running their last focus group for adolescents with adrenal insufficiency.
24th December 2024
Research is a significant priority and source of hope for the leukodystrophy community. Through our research communications and activities, we found that we were able to be an accessible bridge between researchers and individuals in our community.
20th December 2024
19th December 2024
Our Research Summary for December 2024 is ready to view. Our Research Summary includes information about recent leukodystrophy research and clinical trials, including article summaries and direct links to websites and articles.
17th December 2024
We would like to share some really exciting news about the results of the Nexus trial which Minoryx Therapeutics has just released.
11th December 2024
We are pleased to share that the presentations from our Community Weekend 2024 are now available to view on our YouTube channel. We have created a playlist of all the recorded presentations from the event.
3rd December 2024
Our CEO, Sara Hunt, and Support Services Manager, Karen Harrison, attended the ELA International Symposium on Monday 2nd and Tuesday 3rd December 2024. It was an informative two days focusing on ‘Ethics for the benefits of patients’.
28th November 2024
We will be sending our the November edition of our monthly news round-up. It includes charity news, our community and ways to get involved.
25th November 2024
Our Research Summary for November 2024 is ready to view. Our Research Summary includes information about recent leukodystrophy research and clinical trials, including article summaries and direct links to websites and articles.
18th November 2024
The rare disease quality statement project is asking the rare disease community to complete their survey, to develop a set of statements which clearly set out what high-quality, sustainable care, treatment and management look like for rare diseases.
15th November 2024
We have a ‘current clinical and observational trials’ page on our website. The page provides information about current clinical and observational trials for different types of leukodystrophy.
1st November 2024
BridgeBio shares positive data from gene therapy study for Canavan Disease. There has been a continued, progressive improvement in motor function and achievement of motor milestones at 12-months post-treatment.
