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…
Atypical Presentation of MLD Without White Matter Changes
Atypical Presentation of Metachromatic Leukodystrophy (MLD) Without White Matter Changes A recent case study published…
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…
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!
Positive Results Announced for Alexander Disease Clinical Trial
Ionis anounces positive topline results from pivotal study of zilganersen in Alexander disease The Ionis…
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…