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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In Memorial and Honor: March and April 2025

In Memory or In Honor Donation / May 16, 2025

ULF received donations in memorial or honorarium for the following during the months of March and April 2025. We apologize for any inadvertent omittance or misspellings. Please notify us if we have made a mistake at office@ulf.org Honoring Loved Ones Through Generosity We are deeply grateful for the generous donations made in honor and in …

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The Translational Landscape of Reactive Astrocytes Reveals the Impact of eIF2B-mediated Dysregulation in VWM Disease

Research / April 9, 2025

Understanding Vanishing White Matter Disease Through Astrocyte Translation Regulation This study investigates the molecular underpinnings of Vanishing White Matter Disease (VWMD), a severe, inherited neurodegenerative disorder caused by mutations in the eIF2B complex, a critical regulator of mRNA translation and the integrated stress response. VWMD primarily affects astrocytes, the brain’s support cells, yet the specific …

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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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Atypical Presentation of MLD Without White Matter Changes

Research / March 7, 2025

Atypical Presentation of Metachromatic Leukodystrophy (MLD) Without White Matter Changes A recent case study published in Neurology International “Metachromatic Leukodystrophy Presenting with Multiple Cranial Nerve and Lumbosacral Nerve Root Enhancement Without White Matter Changes” highlights an unusual presentation of Metachromatic Leukodystrophy (MLD), a rare autosomal recessive disorder characterized by demyelination in both the central and …

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In Memorial and Honor: February 2025

In Memory or In Honor Donation / March 6, 2025

ULF received donations in memorial or honorarium for the following during the month of February 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 Daniel Nathan Horton In Loving Memory of Daniel. Daxton Wallace Sandra Wallace Deandre …

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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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