New Global Guidelines Issued for Vanishing White Matter (VWM) — A Significant Step Forward for Families and Care Teams We’re pleased to share a major advancement in the care of VWM. A new international consensus document has just been published, offering evidence- and expert-based recommendations for the diagnosis and clinical management of VWM. This is …
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GliaConnect has launched a new global patient registry for individuals and families affected by microgliopathies such as ALSP, BANDDOS, and Nasu-Hakola Disease. In partnership with Sanford CoRDS, this registry helps bring together patients, families, and researchers to advance understanding and treatment of these rare disorders. The data collected will help identify symptoms, improve care management, …
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Learn more at ClinicalTrials.gov!
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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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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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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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 …
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 …
FDA Approves Ctexli (Chenodiol) for Treatment of Cerebrotendinous Xanthomatosis (CTX) Read More »
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 …
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 …
