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…
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
The study publicshed in the New England Journal of Medicine titled “Hematologic Cancer after Gene…
Fall 2024 Newsletter
The Fall 2024 ULF Newsletter has been posted! Find it here
Experiences of mothers of long-term surviving patients with cerebral adrenoleukodystrophy: a qualitative study
Experiences of mothers of long-term surviving patients with cerebral adrenoleukodystrophy: a qualitative study Overview: The…
Gene therapy shows long-term benefit for patients with Cerebral adrenoleukodystrophy (CALD)
Gene therapy shows long-term benefit for patients with Cerebral adrenoleukodystrophy (CALD) Overview: In a study…
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…
In Memorial and Honor: August – September 2024
ULF received donations in memorial or honorarium for the following during the months of August…
PMD Clinical Study
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356…