ASTRO-AXD STUDY IIONIS is inviting people living with Alexander Disease and who are starting or are already being treated with Zanvastro (Zilganersen) and their caregivers to participate in a voluntary observational study. Please review the Astro-AxD Study information for more details Click Here for Full Press Release
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Researchers at the ALC discover a new leukodystrophy From Amsterdam Leukodystrophy Center Newsletter September 2026 Researchers at the ALC, working with international partners, have discovered a new leukodystrophy. So far, the disease has been described in three patients. Click here for full article
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JLR Research Article: Spatial and temporal brain biodistribution of neuropathogenic sphingolipids of Krabbe disease Authors: Tingting Yan, Shih-Chang Hsueh , Salma Begum , Narayana Chelakkara Venkiteswaran, Justin Ellenburg, Boone M. Prentice, and Gustavo H. B. Maegawa Click Here for Full Article
iScience Article: Sphingolipid-neutralizing molecular thereapy reduces psychosine cytotoxicity in Krabbe Disease Authors: Salma Begum, Shin-Change Hsueh, Eara M.Y.Cheria, ….,Michael H. Gelb, Change-Chun Ling, Gustavo H.B. Maegawa Click Here for Full Article
JOURNAL NEWS article: Mapping toxic lipid buildup in a rare neurological disease By Stella Ekaputri Click Here to View Full Article
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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
Research Summaries December 2025 Read More »
The United Leukodystrophy Foundation (ULF) is proud to announce the award of a $33,000 research grant to Dr. Jun Xie, PhD, of UMass Chan Medical School, to support an innovative preclinical gene therapy project focused on Hypomyelination with Atrophy of the Basal Ganglia and Cerebellum (H-ABC). About the Funded Project Project Title:AAV-mediated gene silencing for
ULF Awards $33,000 Research Grant to Advance Gene Therapy for H-ABC Read More »
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
Research Summaries October 2025 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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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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