Stay up to date
16th May 2025
Our Research Summary for May 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.
6th May 2025
The UK National Screening Committee (UK NSC) has today opened a consultation on evidence relating to newborn screening for metachromatic leukodystrophy (MLD).
1st May 2025
Ionis have recently added a new recruitment site to the ORBIT study in the United States (The Children’s Hospital of Philadelphia – CHOP).
30th April 2025
Reconstruction and Computational Modelling for Inherited Metabolic Diseases is a European project involving 34 centres specialising in metabolic diseases from 14 countries.
29th April 2025
Our April 2025 edition is available. This month we will be sharing charity updates, activities for our community to be involved in and ways you can support Alex TLC.
22nd April 2025
Our Research Summary for April 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 April 2025
Don’t forget that presentations from our Community Weekend 2024 are available to view on our YouTube channel. The playlist includes presentations from across the weekend.
16th April 2025
The Rare Disease Quality Statement Project is asking the rare disease community to complete a survey to help finalise a set of statements that define what high-quality, sustainable care, treatment, and management should look like for rare diseases.
14th April 2025
An interesting article exploring the work of founders of rare disease organisations. Our CEO and founder, Sara, contributed her own experience as part of this article.
11th April 2025
Global Leukodystrophy Initiative is working to propel research on leukodystrophies toward clinical trial readiness. This includes a gene therapy for the treatment of MLD.
9th April 2025
Mayo Clinic launches landmark study on rare neurological disease. At the center of this effort is autosomal dominant leukodystrophy (ADLD).
28th March 2025
CALYX is a new clinical trial in cALD by Minoryx Therapuetics. This trial could be suitable if you are an adult male with AMN or cALD.
