Month: February 2025

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 …

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

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 …

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

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 …

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

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 [email protected] 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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