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 …
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 …
The Hidden Impact of Vanishing White Matter (VWM) on Families Read More »
Johana and ella In 2015, the teachers at our eldest’s daughter’s high school noticed she was struggling with remembering things. Johana was 16 and had always experienced learning difficulties. However, the teachers were concerned, so we took her to see a pediatric neurologist. In early January 2016, after an MRI and blood and urine tests, …
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 …
Minoryx’s Leriglitazone MAA for cALD Validated by EMA Read More »
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 …
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 …
