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…
Accumulation of ether phospholipids in induced pluripotent stem cells and oligodendrocyte‐lineage cells established from patients with Sjögren‐Larsson syndrome
The study “Accumulation of Ether Phospholipids in Induced Pluripotent Stem Cells and Oligodendrocyte-Lineage Cells Established…
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,…
Human iPSC-derived myelinating organoids and globoid cells to study Krabbe disease
The article, “Human iPSC-derived myelinating organoids and globoid cells to study Krabbe disease”, explores innovative…
A Special Year-End Message from Dr. Mousumi Bose
My name is Mousumi Bose, and I am the mother of Ilan Betzer. Ilan was born…
Help Us Bring Hope to Families Like Isabella’s This Holiday Season
I’m the aunt of my beautiful niece, Isabella, who was diagnosed with Vanishing White Matter…
Beverly’s Journey of Hope & Giving Back
“I was so fortunate to have been able to attend the ULF conference without worrying…