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…
Research Summaries October 2025
ALEX TLC (Alex, The Leukodystrophy Charity) produces monthly research summaries highlighting recent leukodystrophy research and…
New Global Patient Registry for MicrogliopathiesGlia
GliaConnect has launched a new global patient registry for individuals and families affected by microgliopathies…
New Research: Endogenous Repair in Vanishing White Matter
A newly published article, “Endogenous Repair in Vanishing White Matter” explores how the brain may…
Calyx: New Clinical Trial in cALD
Learn more at ClinicalTrials.gov!
In Memorial and Honor: May – September 2025
ULF received donations in memorial or honorarium for the following during May through September 2025….
Teagan’s Story
A Mother’s Love, A Child’s Legacy: Teagan’s Story Our daughter, Teagan Ann, will forever be…
Positive Results Announced for Alexander Disease Clinical Trial
Ionis anounces positive topline results from pivotal study of zilganersen in Alexander disease The Ionis…
Kim’s Story
Kim’s Story: A Family Bond with ALSP In 2019, my brother Jeffrey Cade was 42 when…
Moa’s Story
Moa’s Story Moa is a fantastic girl with a big, bright smile that charms every…
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…
Johana’s and Ella’s Story
Johana and ella In 2015, the teachers at our eldest’s daughter’s high school noticed she…
Minoryx’s Leriglitazone MAA for cALD Validated by EMA
EMA Validates Minoryx’s Marketing Application for Leriglitazone to Treat cALD Minoryx Therapeutics, a clinical-stage biotech…
In Memorial and Honor: March and April 2025
ULF received donations in memorial or honorarium for the following during the months of March…
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…
ALEXANDER DISEASE CAREGIVER Information
Just Worldwide is conducting a market research study on behalf of a pharmaceutical company. The…
Atypical Presentation of MLD Without White Matter Changes
Atypical Presentation of Metachromatic Leukodystrophy (MLD) Without White Matter Changes A recent case study published…
In Memorial and Honor: February 2025
ULF received donations in memorial or honorarium for the following during the month of February…
FDA Approves Ctexli (Chenodiol) for Treatment of Cerebrotendinous Xanthomatosis (CTX)
Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX) Mirum Pharmaceuticals…