News

Positive Results Announced for Alexander Disease Clinical Trial

Clinical Trials, News / September 22, 2025

Ionis anounces positive topline results from pivotal study of zilganersen in Alexander disease The Ionis Zilganersen Team shared a message with the Alexander disease community: September 22, 2025 Dear Alexander disease community, Ionis announced today positive initial results from the pivotal clinical trial (NCT: 04849741)1 of zilganersen (ION373) in people with Alexander disease (AxD). In …

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The Hidden Impact of Vanishing White Matter (VWM) on Families

News, Research / September 4, 2025

New Research Sheds Light on the Burden for Parents, Siblings, and Partners A groundbreaking international study has revealed something our community has long known: Vanishing White Matter (VWM) doesn’t just affect patients. It deeply impacts their families too. A new study, “The impact of vanishing white matter on unaffected family members,” published in the Orphanet …

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Minoryx’s Leriglitazone MAA for cALD Validated by EMA

News / July 25, 2025

EMA Validates Minoryx’s Marketing Application for Leriglitazone to Treat cALD Minoryx Therapeutics, a clinical-stage biotech company focused on orphan central nervous system (CNS) diseases, has announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for leriglitazone (NEZGLYAL®). This milestone marks an important regulatory step toward approval of a first-in-class oral …

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ALEXANDER DISEASE CAREGIVER Information

News / March 11, 2025

Just Worldwide is conducting a market research study on behalf of a pharmaceutical company. The study aims to gather insights from caregivers of people with Alexander Disease to better understand their experiences and opinions. The research involves a 40-minute online survey. Participants who complete the survey will receive $100 as compensation for their time. If …

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FDA Approves Ctexli (Chenodiol) for Treatment of Cerebrotendinous Xanthomatosis (CTX)

News / February 24, 2025

Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX) Mirum Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) tablets as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare and progressive genetic disorder that affects cholesterol metabolism. CTX leads to harmful cholesterol buildup in …

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The ULF Board of Directors’ statement regarding the changes to healthcare and research because of reduced NIH funding.

News / February 24, 2025

The United Leukodystrophy Foundation Board of Directors’ statement regarding the changes to healthcare and research because of reduced NIH funding. February 24, 2025 “The United Leukodystrophy Foundation (ULF) is deeply concerned about proposed changes in healthcare and research. These changes may limit patient access to life-saving treatments; may impact the ability of physicians and other …

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STUDY: Rescue of in vitro models of CSF1R-related adult-onset leukodystrophy by iluzanebart: mechanisms and therapeutic implications of TREM2 agonism

News, Research / February 19, 2025

Understanding CSF1R-Related Leukodystrophy and a Potential New Treatment: Iluzanebart This study titled Rescue of in vitro models of CSF1R-related adult-onset leukodystrophy by iluzanebart: mechanisms and therapeutic implications of TREM2 agonism and published by Journal of Neuroinflammation, explores CSF1R-related adult-onset leukodystrophy with axonal spheroids and pigmented glia (CSF1R-ALSP), a rare neurodegenerative disease caused by mutations in …

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FDA Approves First Treatment for CTX, a Rare Lipid Storage Disease

News, Research / February 21, 2025

PRESS RELEASE: FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease The U.S. Food and Drug Administration (FDA) has approved Ctexli (chenodiol) as the first treatment for cerebrotendinous xanthomatosis (CTX), a rare lipid storage disorder. CTX is a genetic condition caused by mutations in the CYP27A1 gene, leading to improper cholesterol breakdown …

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STUDY: The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy

News, Research / February 19, 2025

Understanding TUBB4A-Related Leukodystrophy in Children: A Study on Disease Progression and Symptoms This study titled The natural history of variable subtypes in pediatric-onset TUBB4A-related leukodystrophy and published in Molecular Genetics and Metabolism, focuses on TUBB4A-related leukodystrophy, a rare genetic disorder that affects the brain’s white matter (the part of the brain responsible for communication between …

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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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