Research Summaries October 2025
ALEX TLC (Alex, The Leukodystrophy Charity) produces monthly research summaries highlighting recent leukodystrophy research and…
New Research: Endogenous Repair in Vanishing White Matter
A newly published article, “Endogenous Repair in Vanishing White Matter” explores how the brain may…
The Hidden Impact of Vanishing White Matter (VWM) on Families
New Research Sheds Light on the Burden for Parents, Siblings, and Partners A groundbreaking international…
The Translational Landscape of Reactive Astrocytes Reveals the Impact of eIF2B-mediated Dysregulation in VWM Disease
Understanding Vanishing White Matter Disease Through Astrocyte Translation Regulation This study investigates the molecular underpinnings…
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
BridgeBio Shares Positive Data from Canavan Disease Study
BridgeBio Shares Positive Data from High Dose Cohort of Phase 1/2 CANaspire Study of Gene…
A Family’s Journey with ALD – Robert & Nancy
Robert & Nancy: A Family’s journey with ALD When a family faces a leukodystrophy diagnosis,…
New Global Guidelines Issued for Vanishing White Matter (VWM)
New Global Guidelines Issued for Vanishing White Matter (VWM) — A Significant Step Forward for…
New Global Patient Registry for MicrogliopathiesGlia
GliaConnect has launched a new global patient registry for individuals and families affected by microgliopathies…
Calyx: New Clinical Trial in cALD
Learn more at ClinicalTrials.gov!