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…
The ULF Board of Directors’ statement regarding the changes to healthcare and research because of reduced NIH funding.
The United Leukodystrophy Foundation Board of Directors’ statement regarding the changes to healthcare and research…
STUDY: Rescue of in vitro models of CSF1R-related adult-onset leukodystrophy by iluzanebart: mechanisms and therapeutic implications of TREM2 agonism
Understanding CSF1R-Related Leukodystrophy and a Potential New Treatment: Iluzanebart This study titled Rescue of in…
FDA Approves First Treatment for CTX, a Rare Lipid Storage Disease
PRESS RELEASE: FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease The…
STUDY: The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy
Understanding TUBB4A-Related Leukodystrophy in Children: A Study on Disease Progression and Symptoms This study titled…
Study: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes
Research Summary: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes…
28th ASGCT Annual Meeting- Empowering Patients 2025: A Cell and Gene Therapies Summit
Hosted by American Society of Gene + Cell Therapy Learn More This inaugural virtual event March…