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…
In Memorial and Honor: January 2025
ULF received donations in memorial or honorarium for the following during the month of January…
In Memorial and Honor: October – December 2024
ULF received donations in memorial or honorarium for the following during the months of October…
Study: Progression of Spinal Cord Disease in Adult Men With ALD
The article, Progression of Spinal Cord Disease in Adult Men with Adrenoleukodystrophy, details the results…
Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile MLD: a long-term follow-up pilot study
The study “Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a…
A Legacy of Hope: Join Bob & Yvonne in Supporting the ULF
We are Bob and Yvonne Rauner. We are the parents of our two sons, Kevin…
Analysis of GFAP Variants in UK Biobank Suggests Underdiagnosis or Incomplete Penetrance of Adult-Onset Alexander Disease
The article “Analysis of GFAP Variants in UK Biobank Suggests Underdiagnosis or Incomplete Penetrance of…
Finding Strength in Community: Jen’s Story
I’m Jen Martin-Cutler, proud mom to Clark, my handsome ginger son diagnosed with Pelizaeus-Merzbacher Disease (PMD)…
Vanishing White Matter Disease in Children: An Unusual Association, a Novel Mutation, and a Literature Review
The article “Vanishing White Matter Disease in Children: An Unusual Association, a Novel Mutation, and…
A novel homozygous PSAP mutation identified by whole exome sequencing in a consanguineous family with metachromatic leukodystrophy: a case report
The article “A novel homozygous PSAP mutation identified by whole exome sequencing in a consanguineous…
Living with ALD: A Journey of Strength and Community
My name is Margaret Halter. My husband, John, was diagnosed with Adrenoleukodystrophy (ALD)/Adrenomyeloneuropathy (AMN) in 1989…