Ionis Astro-AxD Study
ASTRO-AXD STUDY IIONIS is inviting people living with Alexander Disease and who are starting or…
Researchers at the ALC discover a new leukodystrophy
Researchers at the ALC discover a new leukodystrophy From Amsterdam Leukodystrophy Center Newsletter September 2026Researchers…
JLR Research Article: Spatial and temporal brain biodistribution of neuropathogenic sphingolipids of Krabbe disease
JLR Research Article: Spatial and temporal brain biodistribution of neuropathogenic sphingolipids of Krabbe disease Authors:…
iScience Article: Sphingolipid-neutralizing molecular thereapy reduces psychosine cytotoxicity in Krabbe Disease
iScience Article: Sphingolipid-neutralizing molecular thereapy reduces psychosine cytotoxicity in Krabbe Disease Authors: Salma Begum, Shin-Change…
Research Summaries December 2025
ALEX TLC (Alex, The Leukodystrophy Charity) produces monthly research summaries highlighting recent leukodystrophy research and…
Positive Results Announced for Alexander Disease Clinical Trial
Ionis anounces positive topline results from pivotal study of zilganersen in Alexander disease The Ionis…
Minoxry Therapeutics: Leriglitazone has met the primary endpoint in NEXUS, the pivotal trial for pediatric patients with cALD
Press Release Summary (December 11, 2024): Minoryx Therapeutics, a late-stage biotech company, and Neuraxpharm Group,…
Viking Therapeutics Announces Results from Phase 1b Clinical Trial of VK0214 in Patients with X-ALD
Ionis Astro-AxD Study
ASTRO-AXD STUDY IIONIS is inviting people living with Alexander Disease and who are starting or…
2026 ULF Moser Service Award: Aurora Pujol, MD, PhD
At the United Leukodystrophy Foundation’s 2026 Scientific Symposium & Family Conference, ULF was honored to…
Paxton’s Story: My Angel, My Heart, My Everything
Paxton’s Story: My Angel, My Heart, My Everything My name is Seanna, and my whole…
Manaka’s Journey: Finding Hope in the Unknown
Manaka’s Journey: Finding Hope in the Unknown My name is Megumi, and my daughter Manaka…