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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ALEX TLC (Alex, The Leukodystrophy Charity) produces monthly research summaries highlighting recent leukodystrophy research and clinical trials, with a focus on key findings and advancements in treatment. These summaries are prepared by volunteer Biomedical Science students, who provide their interpretations of the latest studies and findings — helping make complex research more accessible to the …
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, …
New Global Patient Registry for MicrogliopathiesGlia Read More »
A newly published article, “Endogenous Repair in Vanishing White Matter” explores how the brain may attempt to repair the white-matter damage characteristic of Vanishing White Matter (VWM) disease. This groundbreaking study was made possible through brain donations from VWM patients and their families. Their extraordinary generosity enables scientists to deepen their understanding of VWM and …
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Learn more at ClinicalTrials.gov!
ULF received donations in memorial or honorarium for the following during May through September 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 memory of cherished …
A Mother’s Love, A Child’s Legacy: Teagan’s Story Our daughter, Teagan Ann, will forever be the strongest person I have ever known. She was born on November 13th, 2016. A perfectly healthy, beautiful baby girl. Ten fingers and ten toes. Our little miracle. Time went by so quickly, and Teagan was growing into the most …
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 …
Positive Results Announced for Alexander Disease Clinical Trial Read More »
Kim’s Story: A Family Bond with ALSP In 2019, my brother Jeffrey Cade was 42 when he awoke one morning unable to speak clearly. Jeffrey immediately called our family, and we urged him to drive to the hospital. After an overnight stay, the doctors assumed Jeffrey had a minor stroke and asked him to follow up …
Moa’s Story Moa is a fantastic girl with a big, bright smile that charms every soul she meets. A girl with her heart in the right place and a twinkle in her eyes. She leaves no one untouched. We are from Sweden and live in Malmö. Moa is nine years old and lives with her …
