Study: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes

News, Research / February 19, 2025

Research Summary: Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes In this study, titled Impaired Peroxisomal Beta-Oxidation in Microglia Triggers Oxidative Stress and Impacts Neurons and Oligodendrocytes, and publshed by Frontiers in Molecular Neuroscience, investigates how dysfunction in peroxisomes — small cell structures that break down fatty acids — affects …

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28th ASGCT Annual Meeting- Empowering Patients 2025: A Cell and Gene Therapies Summit

News / February 19, 2025

Hosted by American Society of Gene + Cell Therapy This inaugural virtual event March 12 and 13, 2025 (9 a.m. – 12:30 p.m. ET on both days), will provide timely education and engaging discussions on cell and gene therapies (CGTs). In alignment with ASGCT’s strategic plan, the goal is to empower advocates, patients, and caregivers who are …

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In Memorial and Honor: January 2025

In Memory or In Honor Donation / February 18, 2025

ULF received donations in memorial or honorarium for the following during the month of January 2025. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org or by calling 815-748-3211 In honor of Bob Hodge Jr., Brian Greene, Garner Hicks, and Tom Cunningham Sallie Griffiths Daxton …

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In Memorial and Honor: October – December 2024

In Memory or In Honor Donation / January 24, 2025

ULF received donations in memorial or honorarium for the following during the months of October through December 2024. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org or by calling 815-748-3211 In honor of Adam Sledzik Stanislaw Sledzik CTX is very bad ,rare disease. Adrianna …

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Study: Progression of Spinal Cord Disease in Adult Men With ALD

Research / January 22, 2025

The article, Progression of Spinal Cord Disease in Adult Men with Adrenoleukodystrophy, details the results of a long-term study on the progression of spinal cord disease in adult men with ALD. The study, which followed 79 patients over seven years, used clinical tools like the EDSS, SSPROM, and 6MWT to measure disease progression. It showed …

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Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile MLD: a long-term follow-up pilot study

Research / January 22, 2025

The study “Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot study” investigates a pioneering treatment for juvenile metachromatic leukodystrophy (MLD), a rare, inherited neurological disorder caused by a deficiency in the ARSA enzyme. This enzyme deficiency leads to the toxic accumulation of sulfatides, causing progressive demyelination (damage …

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A Legacy of Hope: Join Bob & Yvonne in Supporting the ULF

Share Your Story / December 3, 2024

We are Bob and Yvonne Rauner. We are the parents of our two sons, Kevin and Paul, who had adrenoleukodystrophy and adrenomyeloneuropathy. Our story began with all the same challenges ALD families faced at the time. We navigated a three-year diagnostic odyssey with Kevin. He was “diagnosed” with ADHD and treated for it but yet …

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Analysis of GFAP Variants in UK Biobank Suggests Underdiagnosis or Incomplete Penetrance of Adult-Onset Alexander Disease

Research / December 18, 2024

The article “Analysis of GFAP Variants in UK Biobank Suggests Underdiagnosis or Incomplete Penetrance of Adult-Onset Alexander Disease” investigates the prevalence and clinical significance of mutations in the GFAP gene, which are linked to Alexander disease. This rare neurodegenerative disorder affects the white matter of the central nervous system, leading to a progressive decline in …

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Finding Strength in Community: Jen’s Story

Share Your Story / November 27, 2024

I’m Jen Martin-Cutler, proud mom to Clark, my handsome ginger son diagnosed with  Pelizaeus-Merzbacher Disease (PMD) in December 2015 at 11 months old. Clark’s leukodystrophy journey began with challenges like delayed milestones, difficulty swallowing, and nystagmus, leading us to specialists at Lurie Children’s Hospital. His care team included experts in genetics, pulmonology, neurology, ENT, and a …

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Vanishing White Matter Disease in Children: An Unusual Association, a Novel Mutation, and a Literature Review

Research / December 18, 2024

The article “Vanishing White Matter Disease in Children: An Unusual Association, a Novel Mutation, and a Literature Review” explores Vanishing White Matter Disease (VWMD), a rare genetic disorder that affects the brain’s white matter. This study presents a case involving a novel genetic mutation, contributing to the understanding of the disease’s complexity. VWMD is caused …

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