8th October 2025
We are attending Manchester Rare Disease Showcase
Our Research Analyst, Kristina, will be attending Beacon’s Manchester Rare Disease Showcase on 26th November.
Stay up to date
8th October 2025
Our Research Analyst, Kristina, will be attending Beacon’s Manchester Rare Disease Showcase on 26th November.
29th September 2025
Our next online community meeting will be taking place on Tuesday 21st October 11:30-13:00 (BST). The meeting is open to anyone affected by leukodystrophy.
27th September 2025
Karen presented at the ‘Leukodystrophies: Novel perspectives on treatments and disease monitoring’ course in Paris held by Professor Fanny Mouchel and attended by many leukodystrophy specialists.
26th September 2025
Families being offered maternity care at Sunderland Royal Hospital and South Tyneside District Hospital are among those being invited to take part in the Generation Study.
26th September 2025
Our September 2025 edition is available. This month we will be sharing about Leukodystrophy Awareness Month, Twickenham Stadium Zip Wire, using easyfundraising on the Prime Big Deal Days, two press releases from Ionis and much more.
24th September 2025
Ionis Pharmaceuticals have announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD).
24th September 2025
GPs across England to take ‘3 strikes and rethink approach’ after 3 appointments – as Jess’s Rule rolled out nationwide to prevent avoidable deaths.
22nd September 2025
Ionis have shared a recent press release highlighting the 10 year anniversary of their Surf Away+ event.
22nd September 2025
Thank you to everyone who attended our Community Weekend 2025!
18th September 2025
We are sharing our community for September of a community member who is asking for recommendations for a plane seat cushion.
17th September 2025
Our Research Summary for September 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.
11th September 2025
Have you accessed NHS Wheelchair Services? Share your experience to add to Whizz Kidz’s research